The “Back Story” of the JAMA Wellness Smackdown (Part 1)
This article became the single most influential article in Health Affairs history, with 935 academic citations alone, plus an untold number of start-ups, corporate program implementations, and references in lay publications. Unfortunately, when you attract that much attention with a finding that is basically fabricated, someone is bound to notice. In this case, the someone was me. (I also encouraged RAND’s ace wellness researcher, Soeren Mattke, to take part in the effort, which he expertly did.)  It turns out that most if not all of the studies in this meta-analysis never should have made it t...
Source: The Health Care Blog - April 29, 2019 Category: Consumer Health News Authors: Christina Liu Tags: Health Policy Al Lewis Wellness Workplace Wellness workplace wellness programs Source Type: blogs

Chromosomally speaking, what do you know about sex? Take a quiz to find out.
Women have two X chromosomes (XX) and men have one X and one Y (XY), right? Not always, as you’ll learn from the quiz below. Men can be XX and women can be XY. And many other combinations of X and Y are possible. NIGMS Director’sEarly-Career Investigator LectureSex-Biased Genome Evolution Melissa A. Wilson, Ph.D.Arizona State University Wednesday, April 10, 201910:00-11:30 a.m. ET Lecture followed by Q&A sessionInfo on the ECI Lecture webpage You can learn more by listening to the live stream of a talk, titled “Sex-Biased Genome Evolution,” at 10 a.m. ET on April 10. The speaker,...
Source: Biomedical Beat Blog - National Institute of General Medical Sciences - April 3, 2019 Category: Research Authors: Matt Mills Tags: Being a Scientist Genes Chromosomes Genetics Genome Genomics Source Type: blogs

TXA a Late Bloomer in Bleeding Management
​Tranexamic acid (TXA) was invented by a Japanese husband-and-wife research team in the 1960s. Years earlier, this same research team had discovered epsilon-aminocaproic acid, a derivative and an analogue of the amino acid lysine. In their search for a more potent antifibrinolytic agent, they discovered tranexamic acid, a synthetic analog of the amino acid lysine. Tranexamic acid is eight to 10 times more powerful than epsilon-aminocaproic acid.The antifibrinolytic actions of TXA result from the binding of four or five lysine receptor sites on plasminogen. This binding prevents plasmin from binding to and degrading fibri...
Source: M2E Too! Mellick's Multimedia EduBlog - February 1, 2019 Category: Emergency Medicine Tags: Blog Posts Source Type: blogs

A Tale of 2 FDAs
By ANISH KOKA Frances Oldham Kelsey by all accounts was not mean to have a consequential life.  She was born in Canada in 1914, at a time women were meant to be seen and not heard.  Nonetheless, an affinity for science eventually lead to a masters in pharmacology from the prestigious McGill University.  Her first real break came after she was accepted for PhD level work in the pharmacology lab of a professor at the University of Chicago.  An esteemed professor was starting a pharmacology lab and needed assistants, and the man from Canada seemed to have a perfect resume to fit.  That’s right, ...
Source: The Health Care Blog - June 4, 2018 Category: Consumer Health News Authors: anish_koka Tags: Uncategorized Source Type: blogs

Would You Want To Know Whether You ’re At Risk For Alzheimer’s?
Do genetic tests help in preparing for potential future health issues or open Pandora’s box full of concerns, worries and hypochondriac thoughts? Would you want to know your genetic fate? Whether you are at risk for Alzheimer’s or a chronic disease 30 years in advance? Would you want to live with this kind of information? Would you take the BRCA test to find out that you are at risk for breast cancer? What would you do if you were? The Medical Futurist team contemplated situations requiring hard, life-altering decisions. What would you do? Our genetic heritage carries secrets that are difficult to process In Se...
Source: The Medical Futurist - April 28, 2018 Category: Information Technology Authors: nora Tags: Bioethics Genomics alzheimer disease DNA dna testing doctor-patient doctor-patient relationship DTC future genetics Huntington's patient empowerment personal genomics Source Type: blogs

Interview with Devyn Smith PhD, COO of Sigilon Therapeutics
Sigilon Therapeutics is a Cambridge, MA-based biotech company developing innovative therapeutics by encapsulating cells in a novel coating that renders them invisible to the immune system. The engineered cells contained in the company’s particles can provide long-term continuous therapy for a range of chronic disorders, including hemophilia and diabetes, and eliminate the need for intermittent injection or infusion. With this technological breakthrough Sigilon hopes to “fundamentally change the trajectory of disease treatment.” Medgadget editor Tom Peach recently spoke with Devyn Smith PhD, Chief Strategy...
Source: Medgadget - March 13, 2018 Category: Medical Devices Authors: Tom Peach Tags: Exclusive Genetics Materials Medicine Pediatrics Source Type: blogs

Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant: the researchers infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX –R338L) transgene in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value.They found sustained therapeutic expression of factor IX coagulant activity after gene transfer in the 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophyla...
Source: Clinical Cases and Images - Blog - January 1, 2018 Category: Universities & Medical Training Tags: Hematology NEJM Source Type: blogs

Last of the Seven Summits - Making History for Hemophilia!
Mountaineer and Save One Life board Member Chris Bombardier is set to scale the Last of the Seven Summits, Mt. Vinson, becoming the first person with hemophilia in history to climb all Seven Summits (Source: Disabled World Blogs)
Source: Disabled World Blogs - December 22, 2017 Category: Disability Tags: Blogs - Writings - Stories Source Type: blogs

Funtabulously Frivolous Friday Five 217
LITFL • Life in the Fast Lane Medical Blog LITFL • Life in the Fast Lane Medical Blog - Emergency medicine and critical care medical education blogJust when you thought your brain could unwind on a Friday, you realise that it would rather be challenged with some good old fashioned medical trivia FFFF…introducing Funtabulously Frivolous Friday Five 217: children who changed medicine. Question 1In 1796, What did James Phipps (1788-1853) participate in that significantly changed the course of modern medicine?+ Reveal the Funtabulous Answerexpand(document.getElementById('ddet87874616'));expand(docume...
Source: Life in the Fast Lane - December 8, 2017 Category: Emergency Medicine Authors: Mark Corden Tags: Frivolous Friday Five Christmas disease cow pox Doogie Howser Edward Jenner Haemophilia B IVF James Phipps Lorenzo's oil. Lorenzo Odone Louise Joy Brown small pox stephen christmas test tube baby Source Type: blogs

Ben's Sucky Words
Tomorrow, I ’ll mail to the University of Southern Maine my thesis for my Master of Fine Arts in creative writing degree. Seeing it packaged and ready to go got me thinking about a gift from my first-semester advisor,Suzanne Strempek Shea. She gave me this little notebook to keep with me so my ability to write something is never far from my impulse to.I labeled my pocket-sized notebook Ben Rubenstein ’s Sucky Words to remind me that I have the freedom to write without judgement or consequence. In fact, I must always accept that any of my written work up until its final form sucks. Without that acceptance, I&rsq...
Source: cancerslayerblog - November 26, 2017 Category: Cancer & Oncology Tags: MFA writing/speaking Source Type: blogs

First Electronic Device for Opioid Withdrawal Therapy Approved by FDA
The FDA has given a regulatory green light to the first device that reduces opioid withdrawal symptoms. The NSS-2 Bridge from Innovative Health Solutions, a Versailles, Indiana firm, is stuck to the skin behind the ear and relies on four electrodes that are attached around the ear. The electrodes are used to deliver electric current to a set of occipital and cranial nerves (V, VII, IX, and X), hopefully helping addicts to avoid agitation, insomnia, and other symptoms of kicking opioids. The same device was approved by the FDA three years ago for use in acupuncture and the current approval went under the de novo review...
Source: Medgadget - November 15, 2017 Category: Medical Devices Authors: Editors Tags: Medicine Neurology Pain Management Psychiatry Source Type: blogs

Hi There, I Write Your Stories, I Mean ‘Our Stories’
I write stories about my coworkers. Collectively known as "Our Stories," a new one publishes in my organization's newsletter every other week. Yesterday, the newsletter published a story I wrote about . . . myself. Here is that story with some redactions.* * *Every morning after waking, I prime my body and mind for the day: meditate for 20 minutes, perform 20 pull ­-ups, and then jump on a mini trampoline while singing whatever pops into my head. I then move to the kitchen where I prepare four eggs mixed with hot sauce and grated cheddar on a pan coated with butter, and pour-­over coffee. Once my breakfas...
Source: cancerslayerblog - September 7, 2017 Category: Cancer & Oncology Tags: life lessons writing/speaking Source Type: blogs

Funtabulously Frivolous Friday Five 202
LITFL • Life in the Fast Lane Medical Blog LITFL • Life in the Fast Lane Medical Blog - Emergency medicine and critical care medical education blog Just when you thought your brain could unwind on a Friday, you realise that it would rather be challenged with some good old fashioned medical trivia FFFF…introducing Funtabulously Frivolous Friday Five 202. Question 1 Who is “Cavity Sam”? Clue: You have operated on him on before. + Reveal the Funtabulous Answer expand(document.getElementById('ddet393724755'));expand(document.getElementById('ddetlink393724755')) Cavity Sam is the character o...
Source: Life in the Fast Lane - August 18, 2017 Category: Emergency Medicine Authors: Neil Long Tags: Frivolous Friday Five cavity sam haemophilia male versus female operation rifampicin Royal disease Vitiligo Source Type: blogs

Skin Grafts for Gene Therapy Could Treat Type II Diabetes and Obesity
Scientists at the University of Chicago genetically modified skin cells to produce glucagon-like peptide 1 (GLP1) protein that is beneficial in diabetes and reduces appetite, and these investigators grew the cells in a dish to form a skin graft. The grafts could potentially be used to treat diabetes and obesity, and could provide a new way to perform gene therapy for a variety of diseases. When normal and gene-altered mice ate the high-fat diet — along with varying levels of doxycycline to induce GLP1 release — mice expressing GLP1 (left) gained less weight gain while normal mice (right) grew fat. Credit: ...
Source: Medgadget - August 4, 2017 Category: Medical Devices Authors: Conn Hastings Tags: Genetics Medicine News Source Type: blogs

Needle-Free Viscous Drug Injections: Interview with CEO of Portal Instruments
High viscosity biologic drugs generally require syringe injections, but many patients are extremely uncomfortable around long needles and injection times can create a great deal of anxiety. Portal Instruments, a company out of Cambridge, Massachusetts, has developed a nearly automatic needle-free injector that reminds us of something doctors in science fiction movies would use. We spoke with Patrick Anquetil, CEO of Portal Instruments to find out exactly how a viscous liquid can be made to enter the body without a needle, what this means for the treatment of different diseases, and what additional features the company...
Source: Medgadget - June 30, 2017 Category: Medical Devices Authors: Editors Tags: Exclusive Medicine Pediatrics Source Type: blogs

Top Companies in Genomics
From portable genome sequencers until genetic tests revealing distant relations with Thomas Jefferson, genomics represents a fascinatingly innovative area of healthcare. As the price of genome sequencing has been in free fall for years, the start-up scene is bursting from transformative power. Let’s look at some of the most amazing ventures in genomics! The amazing journey of genome sequencing Genome sequencing has been on an amazing scientific as well as economic journey for the last three decades. The Human Genome Project began in 1990 with the aim of mapping the whole structure of the human genome and sequencing ...
Source: The Medical Futurist - May 30, 2017 Category: Information Technology Authors: nora Tags: Genomics Personalized Medicine AI artificial intelligence bioinformatics cancer DNA dna testing DTC gc3 genetic disorders genetics genome sequencing personal genomics precision medicine Source Type: blogs

Value Frameworks For Rare Diseases: Should They Be Different?
The US health care system is increasingly focusing on value as a basis for reimbursement of pharmacotherapies and devices, and as a result the use of “value frameworks” for measuring and comparing treatment value has grown in recent years. However, the therapies assessed by most frameworks frequently apply to modest-to-large disease populations, rather than the smaller populations affected by rare diseases, where the factors driving value may differ. Rare diseases are different from diseases affecting larger populations in several fundamental ways. In the United States, a rare disease is defined as one that aff...
Source: Health Affairs Blog - April 12, 2017 Category: Health Management Authors: Anupam Jena and Darius Lakdawalla Tags: Costs and Spending Drugs and Medical Innovation Quality Orphan Drug Act Precision Medicine rare disease treatment treatment value value frameworks Source Type: blogs

Patient or Corporate Advocacy Organizations? - New Studies Shed Some Light
This study analyzed public records (US Internal Revenue Service form 990 tax reports, annual reports and website) on the largest US based patient advocacy organizations, that is, those with revenues of at least $7.5 million.  Its goal was to determine how well these organizations disclose conflicts of interests, and how they have COIs, and what policies they have to mitigate their effects.Its main results were that:-Disclosure was modest.  88% of organizations disclosed their donors, 52% disclosed approximate amounts of donations, but only 5% disclosed exact amounts.  74% provided some information about the ...
Source: Health Care Renewal - March 3, 2017 Category: Health Management Tags: conflicts of interest deception health care corruption institutional conflicts of interest patient advocacy groups Source Type: blogs

Chronic Pain and the Opioid Epidemic: Wicked Issues Have No Simple Solutions
Written By Myra ChristopherMy mom was a steel magnolia (i.e., southern and perfectly charming), but she had a steel rod up her back. After her first surgery for stomach cancer at age 53, she refused pain medication because she said that she “could take it.” She was young and strong and committed to “beating cancer.” After nearly two years of chemotherapy, radiation and two more surgeries, the cancer won. Eventually, I watched her beg nurses to give her “a shot” minutes before another was scheduled and be told they were sorry but she would have to wait. I could tell by the expressions on ...
Source: blog.bioethics.net - January 23, 2017 Category: Medical Ethics Authors: Practical Bioethics Tags: Health Care chronic pain Opioid addiction Opioid Epidemic Opioid prescriptions syndicated Source Type: blogs

Get Health Insurance Through Your Employer? ACA Repeal Will Affect You, Too
Much of the recent attention on the future of the Affordable Care Act (ACA) has focused on the fate of the 22.5 million people likely to lose insurance through a repeal of Medicaid expansion and the loss of protections and subsidies in the individual insurance market. Overlooked in the declarations of who stands to lose under plans to “repeal and replace” the ACA are those enrolled in employer-sponsored health plans — the primary source of coverage for people under 65. Job-based plans offered to employees and their families cover 150 million people in the United States. If the ACA is repealed, they stand ...
Source: Health Affairs Blog - January 11, 2017 Category: Health Management Authors: JoAnn Volk Tags: Featured Following the ACA Insurance and Coverage Quality ACA repeal and replace employer-sponsored coverage Employer-Sponsored Insurance Source Type: blogs

The Adventurous are Undergoing Enhancement Gene Therapies
As I've been saying for the past couple of years, gene therapies are straightforward enough and cheap enough to carry out that people are doing it, usually quietly, but it is happening. You only have to be connected enough to know a biotechnologist or two with the right skills, as the example here shows. The stage of the adventurous and the self-experimenters is an important part of the development of any new medical technology, helping to overcome institutional reluctance while gathering initial data on how best to approach such treatments in practice. The next part of the process, something that does requires much greate...
Source: Fight Aging! - January 10, 2017 Category: Research Authors: Reason Tags: Daily News Source Type: blogs

Funtabulously Frivolous Friday Five 170 Christmas Edition
Just when you thought your brain could unwind on a Friday, you realise that it would rather be challenged with some good old fashioned medical trivia FFFF…introducing Funtabulously Frivolous Friday Five 170 Christmas Edition Question 1 Why is Christmas disease so named? + Reveal the Funtabulous Answer expand(document.getElementById('ddet1725772665'));expand(document.getElementById('ddetlink1725772665')) Haemophilia B was first recognized as a different kind of haemophilia in 1952, named after Stephen Christmas, the first patient described with this disease. If that was not festive enough for you then...
Source: Life in the Fast Lane - December 23, 2016 Category: Emergency Medicine Authors: Neil Long Tags: Frivolous Friday Five bowel perforation brussel sprouts Christmas accidents christmas cake decoration Christmas disease Christmas pudding Haemophilia B stephen christmas vitamin k warfarin Source Type: blogs

Reminder: Keep it simple for outpatients
As a clinical student, I’ve been a part of dozens of outpatient clinic visits, but several days ago, I witnessed a clinic visit much unlike the others. For one, our patient arrived not for a 20-minute appointment, but for a three-hour one. As a hemophiliac, this patient came to Stanford once a year, for a comprehensive, coordinated patient care visit, where she saw not only her hematologist but also her social worker, dietitian, nurse coordinator, physical therapist and others. I had the privilege of sitting in on this patient’s entire visit, witnessing the full spectrum of care coordination, and I found myself...
Source: Kevin, M.D. - Medical Weblog - December 1, 2016 Category: Journals (General) Authors: < a href="http://www.kevinmd.com/blog/post-author/hamsika-chandrasekar" rel="tag" > Hamsika Chandrasekar < /a > Tags: Education Primary care Source Type: blogs

Keeping A Finger On The PULSE Of Blood Donation Policy After Orlando
On June 12, 2016, a gunman murdered 49 people at PULSE, a Florida LGBT nightclub, in the most lethal mass shooting in U.S. history. Many more were critically injured, requiring blood transfusions and exacerbating the need for blood donors in the Orlando area. Yet gay, bisexual, and other men who have sex with men (MSM) who wanted to donate blood were turned away. Garrett Jurss, for example, is a gay man who showed up to donate after the urgent call for blood, but was turned away because of the MSM blood policy. “I want to be able to help my brothers and sisters that are out there, that are suffering right now,”...
Source: Health Affairs Blog - September 1, 2016 Category: Health Management Authors: Adam Eickmeyer Tags: Equity and Disparities Featured Quality blood donations DONATE Act FDA HIV/AIDS LGBT Source Type: blogs

EpiPen may still be too cheap
Good stuff, cheap Pick up a newspaper or surf the web and you’ll find story after story taking Mylan to task for EpiPen pricing practices. The list price of a 2-pack has soared from about $100 to $600 over the past decade. The price is deemed too high and the rate of increase is considered particularly unconscionable. Let me offer a brief counterargument: EpiPen is worth the price. A $300 pen regularly rescues children from anaphylactic shock that would otherwise be fatal, offering them the chance to live to 100 instead of dying at 10. (About 20% of patients need a second...
Source: Health Business Blog - August 26, 2016 Category: Health Management Authors: dewe67 Tags: Pharma Policy and politics Source Type: blogs

Hold on. Ready For It? EpiPen May Actually Still Be Too Cheap!!!
By DAVID E. WILLIAMS Pick up a newspaper or surf the web and you’ll find story after story taking Mylan to task for EpiPen pricing practices. The list price of a 2-pack has soared from about $100 to $600 over the past decade. The price is deemed too high and the rate of increase is considered particularly unconscionable. Let me offer a brief counterargument: EpiPen is worth the price. A $300 pen regularly rescues children from anaphylactic shock that would otherwise be fatal, offering them the chance to live to 100 instead of dying at 10. (About 20% of patients need a sec...
Source: The Health Care Blog - August 26, 2016 Category: Consumer Health News Authors: John Irvine Tags: Uncategorized Source Type: blogs

How Patient Groups Have Begun To Influence The Value And Coverage Debate
In 2015, two issues related to medicine could be relied on to generate headlines: drug pricing and the proliferation of new value frameworks that claimed to define the value and even the price of drugs in seemingly easy-to-understand ways. In none of the high-profile skirmishes on pricing or frameworks was the voice or perspective of patients and patient groups very much in evidence. But that is beginning to change, in an evolution of a broader shift in the role that patients are playing in the research and development (R&D) enterprise. A New Culture of Engagement Patients and patient organizations are becoming ever mo...
Source: Health Affairs Blog - June 10, 2016 Category: Health Management Authors: Margaret Anderson and Kristin Schneeman Tags: Costs and Spending Health Professionals Organization and Delivery Quality clinical research patient use of evidence venture philanthropy Source Type: blogs

Really???
In today’s New York Times, embedded in a report about drug companies wooing hemophilia patients, I came across a drug company with the name BIOETHICS ADVANTAGE.  What to say??? (Source: blog.bioethics.net)
Source: blog.bioethics.net - January 14, 2016 Category: Medical Ethics Authors: denasdavis Tags: Health Care syndicated Uncategorized Source Type: blogs

A Year in Review: FDA 2015 New Drug Approvals
The approval of first-of-a-kind drugs rose last year to forty-one, resulting in the highest level of newly approved U.S. drugs in nineteen years. The total number of new drugs approved last year was even higher at sixty-nine. The rising figures reflect an industry-wide desire to research and develop drugs for rare and hard-to-treat diseases. The newly approved drugs serve to advance medical care and the health of patients suffering from many ailments, including various forms of cancer, heart failure, and cystic fibrosis. Additionally, more than 40% of the new therapies were approved for treatment of rare or "orphan&...
Source: Policy and Medicine - January 13, 2016 Category: American Health Authors: Thomas Sullivan - Policy & Medicine Writing Staff Source Type: blogs

Pharmacy Benefit Managers Begin to Cut Ties With Specialty Pharmacies
Conclusion As these cases continue to wind their way through the courts, it is unknown if they will be joined by others. However, what is known is that all pharmacies can likely expect greater oversight after the disclosures of Valeant and Philidor.       Related StoriesMedicare Releases Drug Data to the Public: Medicare Drug Spending Dashboard 2014Health and Human Services Drug Pricing ForumSenators McCain and Grassley Request HHS for Approval of Importation of Certain Drugs  (Source: Policy and Medicine)
Source: Policy and Medicine - January 11, 2016 Category: American Health Authors: Thomas Sullivan - Policy & Medicine Writing Staff Source Type: blogs

Implementing Health Reform: Essential Community Providers; The Contraceptive Coverage Accommodation
The week of August 27, 2015 was quiet in terms of Affordable Care Act implementation activities, but there were at the end of the week on August 21 a couple of noteworthy developments. First, the Centers for Medicare and Medicaid Services published in the Federal Register notice of a new collection of data regarding essential community providers (ECPs) to support the certification of qualified health plans (QHPs) for plan year 2017.  Later in the day, CMS published the data collection forms and supporting statements at its Paperwork Reduction Act website. The ACA requires QHP insurers, including Stand-alone ...
Source: Health Affairs Blog - August 23, 2015 Category: Health Management Authors: Timothy Jost Tags: Equity and Disparities Following the ACA Insurance and Coverage Source Type: blogs

TWiV 350: Viral gene therapy with Katherine High
On episode #350 of the science show This Week in Virology, Vincent speaks with Katherine High about her career and her work on using viral gene therapy to treat inherited disorders. This episode is drawn from one of twenty-six video interviews with leading scientists who have made significant contributions to the field of virology, part of the new edition of the textbook Principles of Virology. You can find TWiV #350 at www.twiv.tv. (Source: virology blog)
Source: virology blog - August 16, 2015 Category: Virology Authors: Vincent Racaniello Tags: This Week in Virology aav adeno-associated virus blindness factor IX gene therapy hemophilia Katherine High Leber's congenital amaurosis monogenetic vector viral Source Type: blogs

Dose recommendations for extended half-life hemophilia factor products fall short
We, like many in the hemophilia community, were excited to see extended half-life (EHL) factor VIII and IX products start coming to market over the last few months. These products — and expected future products — promise equivalent or greater prophylactic bleeding control with fewer infusions, and so could greatly enhance patients’ quality of life. Continue reading ... Your patients are rating you online: How to respond. Manage your online reputation: A social media guide. Find out how. (Source: Kevin, M.D. - Medical Weblog)
Source: Kevin, M.D. - Medical Weblog - July 2, 2015 Category: Journals (General) Authors: Tags: Meds Hematology Medications Source Type: blogs

CMS Releases 2013 Medicare Payment Data for Hospitals and Physicians
  Yesterday, the Centers for Medicare and Medicaid Services (CMS) announced the release of utilization and payment data for both Medicare hospital services (inpatient and outpatient) and for physicians and suppliers. This is the third year the hospital data was released and the second year that the physician and supplier data was released. Indeed, the big troves of healthcare data keep coming. On April 30, CMS published information on 2013 Medicare Part D payments. At the end of this month, on June 30, CMS is scheduled to release the first full year of pharmaceutical and medical device transfers ...
Source: Policy and Medicine - June 2, 2015 Category: American Health Authors: Thomas Sullivan Source Type: blogs

5 things to know about what’s new in hemophilia
From new longer-acting drugs to promising gene therapy trials, much is changing in the treatment of hemophilia, the inherited bleeding disorder in which the blood does not clot. I will mark Hemophilia Awareness Month by discussing research and treatment progress, as well as remaining challenges. 1. Many more treatment products are being introduced, including some that last longer. In people with hemophilia, a “factor” — or blood protein that helps normal clots form — is missing or defective. Of the approximately 20,000 people with hemophilia in the U.S., about 80 percent suffer from hemophilia A, wh...
Source: Kevin, M.D. - Medical Weblog - March 18, 2015 Category: Journals (General) Authors: Tags: Conditions Hematology Source Type: blogs

Drug Price Policy: New Transparency Bill Would Require Drug Companies To Report Costs For High Cost Drugs; Will Annuities Be The Future Payment Model For Expensive Medicine?
An interesting claim against the pharmaceutical industry (though usually made by only industry's most hardened critics) is that companies don’t want to find a cure—they’d prefer lifelong patients. Recently, Gilead Sciences indeed found a cure for hepatitis C--one that both gets rid of the hepatitis C virus in a patient’s body and does so without many of the terrible side effects that plagued previous therapies. Rather than embracing the medical breakthrough, however, many articles focused on the $84,000 price tag for the full round of treatment. Few mentioned the long-term cost savings now that...
Source: Policy and Medicine - February 26, 2015 Category: American Health Authors: Thomas Sullivan Source Type: blogs

Implementing Health Reform: 2016 Benefit And Payment Final Rule, Consumer & Provider Provisions
On February 20, 2015, the Centers for Medicare and Medicaid Services (CMS) of the Department of Health and Human Services published its massive Notice of Benefit and Payment Parameters (BPP rule) for 2016 Final Rule, accompanied by a fact sheet.  This rule addresses a host of issues involving the continuing implementation of the Affordable Care Act for 2016.  A few provisions, however, affect the 2015 year as well and a number of provisions will not be implemented until 2017. The BPP rule amends and updates existing rules; thus, it must be read in tandem with rules that have been promulgated earlier, which a...
Source: Health Affairs Blog - February 22, 2015 Category: Health Management Authors: Timothy Jost Tags: Access All Categories Consumers Disparities Health Reform Insurance Pharma Policy States Source Type: blogs

Remotely and Noninvasively Controlling Genes and Cells in Living Animals
Researchers are developing a system to remotely control genes or cells in living animals with radio wave technology similar to that used to operate remote control car keys. Credit: Stock image. One of the items on biomedical researchers’ “to-do” list is devising noninvasive ways to control the activity of specific genes or cells in order to study what those genes or cells do and, ultimately, to treat a range of human diseases and disorders. A team of scientists recently reported progress on a new, noninvasive system that could remotely and rapidly control biological targets in living animals . The system...
Source: Biomedical Beat Blog - National Institute of General Medical Sciences - February 13, 2015 Category: Research Authors: Srivalli Subbaramaiah Tags: Chemistry and Biochemistry Genetics Source Type: blogs

Research and Reviews in the Fastlane 052
This study looked at compliance with discharge instructions. Surprisingly (or maybe not so), 39% of pediatric patients returned to play (RTP) on the day of the injury. RTP is widely recognized as a risk for recurrent and more severe concussions as well as significant morbidity. It is the duty of the Emergency Physician to stress the importance of discharge instructions as well as the importance of appropriate follow up. Recommended by: Anand Swaminathan PediatricsSingleton T et al. Emergency department care for patients with hemophilia and von Willebrand disease. J Emerg Med. 2010; 39(2): 158-65. PMID: 18757163 Bleeding...
Source: Life in the Fast Lane - October 9, 2014 Category: Emergency Medicine Authors: Nudrat Rashid Tags: Administration Anaesthetics Cardiology Clinical Research Education Emergency Medicine Haematology Infectious Disease Intensive Care International Emergency Medicine Microbiology Neurosurgery Obstetrics / Gynecology Ophthalmology Source Type: blogs

Physician Payments Sunshine Act: Organizations Respond to CMS
  September 2nd marked the last day for comments on CMS’ proposed rule to eliminate the accredited continuing medical education (CME) exemption from Sunshine Act reporting.  In an overwhelming display of support for the exemption, over 800 comments were submitted encouraging the agency to either maintain or expand the current exclusion. -Total comments supporting maintenance or expansion of the CME exemption:  820 -Total comments supporting elimination of the CME exemption:  approximately 20 -Percentage of comments supporting the CME exemption: 98% We have followed this issue closely, and recentl...
Source: Policy and Medicine - September 8, 2014 Category: American Health Authors: Thomas Sullivan Source Type: blogs

Research and Reviews in the Fastlane 041
This study prospectively validated whether an age-adjusted D-dimer cutoff was associated with an increased diagnostic yield of D-dimer in elderly patients with suspected PE. Compared with a fixed D-dimer cutoff, the combination of pretest clinical probability assessment with age-adjusted D-dimer cutoff was associated with a larger number of patients in whom PE could be considered ruled out with a low likelihood of subsequent clinical venous thromboembolism. So if this is not your clinical practice already, maybe time to use age adjust d-dimer values? Recommended by: Jerremy Fried Read More: Age Adjusted D-Dimer Testing (RE...
Source: Life in the Fast Lane - July 29, 2014 Category: Emergency Medicine Authors: Soren Rudolph Tags: Clinical Research R&R in the FASTLANE critical care Emergency Medicine Intensive Care literature recommendations research and reviews Source Type: blogs

Clot-building nanoparticles raise survival rate following blast trauma Read more: Clot-building nanoparticles raise survival rate following blast trauma
A type of artificial platelet being developed to help natural blood platelets form clots faster offers promise for saving the lives of soldiers, as well as victims of car crashes and other severe trauma.In preclinical tests led by a Case Western Reserve University researcher, the artificial platelets, called "hemostatic nanoparticles," when injected after blast trauma dramatically increased survival rates and showed no signs of interfering with healing or causing other complications weeks afterward."The nanoparticles have a huge impact on survival—not just in the short term, but in the long term,"...
Source: Medical Hemostat - June 30, 2014 Category: Technology Consultants Authors: hemostatguy at gmail.com (hemostat guy) Source Type: blogs

Research and Reviews in the Fastlane 036
This study found that a high percentage (49%) of patients with serious adverse outcomes after an ED visit for COPD were not initially admitted to the hospital. The authors used logistic regression to derive a decision instrument to aid in determining which patients with COPD exacerbation should be admitted based on risk stratification. The study does not show that admission improves outcomes but the instrument may prove useful for risk stratification if it is prospectively validated. Recommended by: Anand Swaminathan Emergency Medicine, Critical care, Anaesthetics Barends CRM ,Absalom AR. Tied up in science: unknotting ...
Source: Life in the Fast Lane - June 26, 2014 Category: Emergency Medicine Authors: Nudrat Rashid Tags: Anaesthetics Emergency Medicine Evidence Based Medicine Featured Health Infectious Disease Intensive Care Respiratory Resuscitation critical care literature R&R in the FASTLANE recommendations research and reviews Source Type: blogs

Drug companies developing longer-acting clotting agents for hemophiliacs
Several drug companies, such as Biogen Idec and Novo Nordisk, are developing new, longer-acting versions of the blood clotting factors used by people with hemophilia. Patients with severe forms of the disease need regular infusions, lasting 30 minutes or more, of relatively short acting and very expensive clotting factors.The new longer-lasting hemophilia B products can be given every 10 days or two weeks, offering significant advantages for patients, especially young children, who now need infusions every two or three days.Hemophilia is hereditary, passed from parent to child through genes. People with hemophilia have lit...
Source: Medical Hemostat - March 23, 2014 Category: Technology Consultants Authors: hemostatguy at gmail.com (hemostat guy) Source Type: blogs

Blood Coagulation Testing Using Smartphone Touchscreens
People who are prone to bleeding due to poor blood clotting, such as those with hemophilia or on anticoagulants, are often required to take blood tests. These test are usually done in clinics and hospitals, adding to the patient’s burden and expense of extra travel just to make sure that blood is adequately anticoagulated. New technology that is being developed at Qloudlab, a startup based at École polytechnique fédérale de Lausanne (EPFL) in Switzerland, uses capabilities already present in today’s smartphone screens for blood coagulation testing. The system works by first applying a micro...
Source: Medgadget - March 18, 2014 Category: Technology Consultants Authors: Editors Tags: Medicine Net News Source Type: blogs

Hepatitis C in Egypt
The following background data on Hepatitis C in Egypt are abstracted from Gideon www.GideonOnline.com and the Gideon e-book series. [1,2] Primary references available on request. Incidence and Prevalence: Hepatitis C is the most common cause of chronic liver disease in Egypt. The nationwide carriage rate in 1997 was estimated at 6 to 8 million, or 18.1% of the population – the highest rate in the world. A study published in 2010 estimated the yearly rate at 500,000 new cases (0.7% of the population); while a study published in 2013 estimated the yearly rate at fewer than 150,000 cases. An analysis published in 2009...
Source: GIDEON blog - December 19, 2013 Category: Databases & Libraries Authors: Dr. Stephen Berger Tags: Ebooks Epidemiology ProMED Egypt hepatitis c Source Type: blogs

Baxter submits application to FDA for pediatric indication of Rixubis to treat Hemophilia B
Baxter International has filed an application to the US Food and Drug Administration (FDA) for a pediatric indication for Rixubis [Coagulation Factor IX (Recombinant)] to treat hemophilia B.The submission was based on a Phase II/III clinical trial, designed to assess the efficacy and safety of Rixubis in 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B.In 2013, the company had secured FDA approval for Rixubis in the US for adults with hemophilia B and it had filed for marketing approval in Europe in November.During the trial, patients were treated with a twice-week...
Source: Medical Hemostat - December 11, 2013 Category: Technology Consultants Authors: hemostatguy at gmail.com (hemostat guy) Source Type: blogs

Pharmalot... Pharmalittle... Good Morning
Rise and shine, everyone. Another busy day is on the way. This is to be expected, though, yes? After all, not much gets accomplished when one is sitting still. Just the same, we are able to enjoy a warm sun hovering over the Pharmalot corporate campus, where the mascots are lounging, the short people are hustling off, as usual, to their houses of learning and another cup of stimulation is brewing in the cafeteria. And this is our signal to get cracking. Meanwhile, here are some items of interest. Hope you have a grand day and keep us in mind if you run across anything interesting... Biosimilars Are Slow To Take Off In Euro...
Source: Pharmalot - December 3, 2013 Category: Pharma Commentators Authors: esilverman Source Type: blogs

Baxter completes patient enrollment in phase III trial of BAX 855, extended half-life rFVIII to treat haemophilia A
Baxter International Inc. has completed enrollment in its phase III clinical trial of BAX 855, an investigational extended half-life, recombinant factor VIII (rFVIII) treatment for haemophilia A. The ongoing trial is aimed at assessing the efficacy of the compound in reducing annualized bleed rates (ABR) in both prophylaxis and on-demand treatment schedules, and will also evaluate its safety and pharmacokinetic profile.BAX 855 was designed based on the full-length ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] molecule, a product with 10 years of real-world experience. The BAX 855 molecule was modi...
Source: Medical Hemostat - November 15, 2013 Category: Technology Consultants Authors: hemostatguy at gmail.com (hemostat guy) Source Type: blogs