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Grand mal seizure shows rare brain disease in child
Jayda Suttie, 10, was diagnosed with vanishing white matter disease when she was nine. Vancouver doctors found out she had the rare disease after she suffered from a grand mal seizure. (Source: the Mail online | Health)
Source: the Mail online | Health - September 18, 2017 Category: Consumer Health News Source Type: news

Rural Hospitals Struggle To Stock Expensive Drugs That Could Save Lives
Rural healthcare providers often struggle to stock sufficient quantities of certain kinds of life-saving prescription drugs because of their high price tag. A provision expanded under the Affordable Care Act extended a discount program for hospitals that primarily serve low-income people to cover rural providers as well. However, this provision includes an exception for "orphan drugs," or prescription medications that treat rare diseases, meaning they are not discounted for rural facilities. Because many prescriptions are granted orphan drug status while they are being tested on rare conditions, this exception pr...
Source: News stories via the Rural Assistance Center - September 15, 2017 Category: Rural Health Source Type: news

Rare disease drugmaker Alexion to move headquarters to Boston
Alexion Pharmaceuticals said Tuesday that it plans to move its headquarters from New Haven, Connecticut to Boston by mid-2018, becoming the latest big drugmaker to make Massachusetts home. Alexion (Nasdaq: ALXN) said that 400 positions would be located in the new headquarters, with around 450 employees remaining in New Haven. A spokeswoman for the company did not immediately respond to a request for comment on the potential location or size of the new Boston office. Alexion already has an 81,000… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - September 12, 2017 Category: Health Management Authors: Max Stendahl Source Type: news

Rare disease drugmaker Alexion to move headquarters to Boston
Alexion Pharmaceuticals said Tuesday that it plans to move its headquarters from New Haven, Connecticut to Boston's Seaport District by mid-2018, becoming the latest big drugmaker to make Massachusetts home. Alexion (Nasdaq: ALXN) said that 400 positions would be located in the new headquarters at 121 Seaport Blvd., with around 450 employees remaining in New Haven. Alexion already has an 81,000 square-foot research and development office in Lexington. The 17-story, elliptical-shaped glass office… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 12, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

Iron deficiency warning: Could YOU spot symptoms of these three RARE diseases?
IRON deficiency is one of the possible causes of a rare disease called pica syndrome where sufferers eat everything from paint to faeces. (Source: Daily Express - Health)
Source: Daily Express - Health - September 9, 2017 Category: Consumer Health News Source Type: news

4-ft woman with rare disease becomes a dancer
Tiffany Geigel, 32, from New York, has Jarco-Levin syndrome, which affects bone growth and has left her battling daily pain and breathing problems from her lungs being crushed. (Source: the Mail online | Health)
Source: the Mail online | Health - September 8, 2017 Category: Consumer Health News Source Type: news

Terminal copper deficiency Menkes film unites families
New York father-of-two Daniel DeFabio created a film about the rare disease his eight-year-old son Lucas (pictured) has, which is called Menkes and affects one in 100,000 children in the US. (Source: the Mail online | Health)
Source: the Mail online | Health - September 7, 2017 Category: Consumer Health News Source Type: news

Abiogen Pharma-R & D, Production, Contract Manufacturing, and Marketing of Pharmaceutical Drugs
Abiogen Pharma is a specialist pharmaceutical company that focuses on bone metabolism and rare diseases. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - September 5, 2017 Category: Pharmaceuticals Source Type: news

Eiger Announces FDA Orphan Drug Designation Granted for Pegylated Interferon Lambda in Hepatitis Delta Virus Infection
PALO ALTO, Calif., Sept. 5, 2017 -- (Healthcare Sales & Marketing Network) -- Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for rare diseases, today announced that the U.S. Food and D... Biopharmaceuticals, FDA Eiger BioPharmaceuticals, Pegylated Interferon Lambda, Hepatitis Delta Virus (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - September 5, 2017 Category: Pharmaceuticals Source Type: news

Artist Creates Eye-Opening Illustrations About Her Rare Disease
"Keep telling your story and eventually someone will listen." (Source: Healthy Living - The Huffington Post)
Source: Healthy Living - The Huffington Post - September 1, 2017 Category: Consumer Health News Source Type: news

Mediaplanet Teams Up with XpertDox and NORD to Explore the Future for...
Mediaplanet’s Rare Diseases campaign, found within USA TODAY and online, helps educate readers on rare diseases.(PRWeb September 01, 2017)Read the full story at http://www.prweb.com/releases/2017/09/prweb14656675.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - September 1, 2017 Category: Pharmaceuticals Source Type: news

Necrotising Ulcerative Gingivitis: A Literature Review
Conclusion: Although there is low prevalence of NUG, its importance should not be underestimated as one of the most severe responses to the oral biofilm. Risk factors must be investigated and addressed. Treatment should consist of gentle superficial debridement, oral hygiene instruction and prescription of mouthwash and antibiotics in severe cases. (Source: Dental Technology Blog)
Source: Dental Technology Blog - August 31, 2017 Category: Dentistry Source Type: news

FDARA: Making a difference for industry and patients
Peter Marks, M.D., Ph.D., Director of FDA’s Center for Biologics Evaluation and Research By: Peter Marks, M.D., Ph.D., Jeffrey Shuren, M.D., J.D., and Janet Woodcock, M.D. For decades, user fees paid by the medical products industry have provided critical resources needed to conduct product reviews in a timely fashion and to help ensure the safety and effectiveness of medical products that American patients depend upon. Since passage of the first medical product user fee act in 1992, the user fee laws and corresponding performance goals and program enhancements have helped evolve the drug and device review process in...
Source: Mass Device - August 22, 2017 Category: Medical Devices Authors: Danielle Kirsh Tags: Blog FDA Voice Source Type: news

An Unexpected Journey
Journey[noun]: The act of travelling from one place to another.By mapping the patient journey – the path taken from diagnosis through treatment to management, recovery or death – pharmaceutical companies are playing an important role in improving patient outcomes.However, the insights emerging from this research can also shape their marketing strategy and activities, says Rebecca Joslin, Head Customer Experience Strategy at Shire.Better understanding of the key touch or pain points across the patient journey allows us to identify opportunities on how we can better support our customers, healthcare professionals...
Source: EyeForPharma - August 21, 2017 Category: Pharmaceuticals Authors: Des Sampson Source Type: news

Joint pain and headache could be warning sign of THIS rare disease
VASCULITIS is a condition which causes inflammation of the blood vessels - triggered by the immune system. (Source: Daily Express - Health)
Source: Daily Express - Health - August 17, 2017 Category: Consumer Health News Source Type: news

Blood biopsy reveals unique, targetable genetic alterations in patients with rare cancer
(University of California - San Diego) Using fragments of circulating tumor DNA in blood, University of California San Diego School of Medicine researchers were able to identify theoretically targetable genetic alterations in 66 percent of patients with cancer of unknown primary (CUP), a rare disease with seven to 12 cases per 100,000 people each year. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 16, 2017 Category: International Medicine & Public Health Source Type: news

Charlie Gard's parents will set up £1.3million foundation
Charlie Gard's parents have announced they will use £1.3 million donated by well-wishers to set up a 'Charlie Gard Foundation' to help other children with rare diseases. (Source: the Mail online | Health)
Source: the Mail online | Health - August 15, 2017 Category: Consumer Health News Source Type: news

Artificial blood vessels mimic rare accelerated aging disease
(Duke University) Biomedical engineers have grown miniature human blood vessels using stem cells taken from patients with an extremely rare genetic disease called progeria that causes symptoms resembling accelerated aging in children. The blood vessels exhibit many of the symptoms and drug reactions associated with progeria and will help doctors and researchers screen potential therapeutics for the disease, and other rare diseases, more rapidly. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - August 15, 2017 Category: International Medicine & Public Health Source Type: news

Atlantic Research Group Forms Rare Disease Strategic Advisory Board to...
New collaboration between patient advocacy groups, government institutions and drug developers to address challenges in rare disease.(PRWeb August 10, 2017)Read the full story at http://www.prweb.com/releases/2017/08/prweb14585611.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - August 10, 2017 Category: Pharmaceuticals Source Type: news

Why this drug company is focusing on rare diseases — BioFlash podcast
ChemoCentryx has seen a Big Pharma partner give drugs back. It went public before biotech IPOs were cool. Now it's looking to score success by pursuing FDA approval in rare kidney diseases. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - August 9, 2017 Category: American Health Authors: Ron Leuty Source Type: news

Albert Einstein College of Medicine and the Children's Hospital at Montefiore Help Lead Genomic Study for Children Underrepresented in Research
August 8, 2017—BRONX, NY—Genomic sequencing holds tremendous potential for improving the way rare diseases are diagnosed and treated. But important questions remain unanswered, such as whether the technology improves health outcomes for patients and how to ensure that diverse populations have equal access to the benefits of genomic sequencing. (Source: Einstein News)
Source: Einstein News - August 8, 2017 Category: Universities & Medical Training Source Type: news

Shire eyes former Ariad building in Cambridge expansion
The rare disease drugmaker is nearing a deal to sublease a 230,000 square-foot space in the heart of Kendall Square, according to real estate industry sources. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 3, 2017 Category: Biotechnology Authors: Max Stendahl Source Type: news

Shire may spin off hyperactivity drugs, raises forecasts
LONDON (Reuters) - Shire, the London-listed pharmaceutical firm built up by acquisitions, said it might spin off its hyperactivity drugs business into a separate company and focus solely on rare disease treatments. (Source: Reuters: Health)
Source: Reuters: Health - August 3, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Pope Francis tweets prayers for parents of Charlie Gard, the baby who died of a rare disease
(Source: ABC News: Health)
Source: ABC News: Health - July 28, 2017 Category: Consumer Health News Tags: Health Source Type: news

GSK Gives Up on Rare Diseases as Gene Therapy Gets Two Customers GSK Gives Up on Rare Diseases as Gene Therapy Gets Two Customers
GlaxoSmithKline is swimming against the tide by getting out of treatments for rare diseases at a time when rivals like Sanofi and Shire see the field as a rich seam for profits.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 28, 2017 Category: Consumer Health News Tags: Family Medicine/Primary Care News Source Type: news

Eiger Announces FDA Fast Track Designation Granted for Pegylated Interferon Lambda in Hepatitis Delta Virus Infection
PALO ALTO, Calif., July 27, 2017 -- (Healthcare Sales & Marketing Network) -- Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for rare diseases, today announced that the U.S. Food and D... Biopharmaceuticals, FDA Eiger BioPharmaceuticals, Hepatitis Delta Virus (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 27, 2017 Category: Pharmaceuticals Source Type: news

Alexion posts higher second-quarter earnings on strong Soliris sales
NEW YORK (Reuters) - Alexion Pharmaceuticals Inc posted a better than expected second-quarter profit on Thursday and raised its full-year forecast on strong sales of its expensive rare disease treatment Soliris. (Source: Reuters: Health)
Source: Reuters: Health - July 27, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Diabetes Crisis Continues To Rage, But Biopharma's Response Is Tepid
However, if you were running an R&D organization, would you use your precious resources to invest in diabetes R&D, or would you look at other important areas like cancer and rare diseases, where the need is great but the ultimate development costs are less? (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 27, 2017 Category: Pharmaceuticals Authors: John LaMattina, Contributor Tags: NYSE:LLY NYSE:GSK NYSE:MRK Source Type: news

GSK gives up on rare diseases as gene therapy gets two customers
LONDON (Reuters) - GlaxoSmithKline is swimming against the tide by getting out of treatments for rare diseases at a time when rivals like Sanofi and Shire see the field as a rich seam for profits. (Source: Reuters: Health)
Source: Reuters: Health - July 26, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Genetic sequencing unravels rare disease mysteries
When Audrey Lapidus ’ 10-month old son, Calvin, didn’t reach normal milestones like rolling over or crawling, she knew something was wrong.“He was certainly different from our first child,” said Lapidus, of Los Angeles. “He had a lot of gastrointestinal issues and we were taking him to the doctor quite a bit.”Four specialists saw Calvin and batteries of tests proved inconclusive. Still, Lapidus persisted.“I was pushing for even more testing, and our geneticist at UCLA said, ‘If you can wait one more month, we’re going to be launching a brand new test called exome sequen...
Source: UCLA Newsroom: Health Sciences - July 18, 2017 Category: Universities & Medical Training Source Type: news

Colorado boy's muscles 'turning to bone'
Caleb Burgess, an 11-year-old from Castle Rock, Colorado, was diagnosed with a rare disease called Fibrodysplasia Ossficans Progressiva (FOP), which is also called 'Stone Man Disease'. (Source: the Mail online | Health)
Source: the Mail online | Health - July 17, 2017 Category: Consumer Health News Source Type: news

Rare disease clues found in cell's recycling system
Researchers with the National Institutes of Health uncovered a compound in a cell's recycling system that could lead to a treatment for a rare genetic disease. (Source: Health News - UPI.com)
Source: Health News - UPI.com - July 17, 2017 Category: Consumer Health News Source Type: news

NIH scientists find rare disease clues in cell ’s recycling system
The work could lead to a new generation of potential therapies for Niemann-Pick type C1 and similar disorders. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - July 17, 2017 Category: American Health Source Type: news

Cambridge BioMarketing joins Ashfield
Cambridge BioMarketing is an industry leader in orphan and rare disease drug launches, a fast-growing area of drug development and commercialisation. The orphan drug and rare disease market is forecasted to grow by a CAGR of 11% between 2017 and 2022 to $209bn, with orphan drugs estimated to account for 21.4% of prescription sales by 20221. Cambridge BioMarketing also has a strong creative and digital function in a market where digital content is becoming increasingly important for pharmaceutical companies. The acquisition of Cambridge BioMarketing provides a platform for growth, specifically in Cambridge, Massachusetts an...
Source: Ashfield Healthcare News - July 17, 2017 Category: Pharmaceuticals Authors: Josee Leach Tags: Ashfield Source Type: news

NIH scientists find rare disease clues in cell's recycling system
(NIH/National Center for Advancing Translational Sciences (NCATS)) Scientists have demonstrated how an investigational drug works against a rare, fatal genetic disease, Niemann-Pick type C1 (NPC1). They found that a closely related compound will activate an enzyme, AMPK, triggering a cellular 'recycling' system that helps reduce elevated cholesterol and other accumulated fats in the brains and livers of NPC1 patients, which are hallmarks associated with severe neurological problems. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 17, 2017 Category: International Medicine & Public Health Source Type: news

British Drugs Body to Challenge New Cost Rules in Court British Drugs Body to Challenge New Cost Rules in Court
Britain's pharmaceuticals trade body plans to go to court to challenge new cost rules for medicines for very rare diseases, describing the measures on Monday as'inappropriate and unworkable'.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 11, 2017 Category: Consumer Health News Tags: Internal Medicine News Source Type: news

Celebrating 10 years of success for Infusion
As the business took off, more space was needed, and the company moved in 2009 to “real” office space just up the road in Haddam, CT, across the street from the Connecticut River, eventually employing a staff of 10 that year. A year later, in 2010, Infusion had doubled in size and became part of the UDG/Ashfield family. Infusion continued to grow at a steady pace into new therapeutic areas with more clients and employees, and in October 2016, Infusion left its country setting and moved into larger corporate offices in downtown Middletown, CT. Our staff now comprises 45 people and includes account executives, me...
Source: Ashfield Healthcare News - July 11, 2017 Category: Pharmaceuticals Authors: Sarah Edwards Tags: Ashfield Source Type: news

How FDA plans to help consumers capitalize on advances in science
By: Scott Gottlieb, M.D. We’re at a point in science where new medical technologies hold out the promise of better treatments for a widening number of vexing conditions. Over the last few decades, science has enabled fundamental advances in our understanding of the genetic and protein bases of human disease. These developments are already being translated into new medicines. In more cases, these treatments target the underlying mechanisms that drive different diseases. These advances hold out the promise of arresting and even curing a growing number of diseases. To build upon such opportunities, FDA will soon unveil ...
Source: Mass Device - July 11, 2017 Category: Medical Devices Authors: Danielle Kirsh Tags: Blog FDA Voice Source Type: news

FDA Reverses Decision, Allowing Amicus To File For Rare Disease Drug
Last November, tiny Amicus Therapeutics said that the Food and Drug Administration would not consider approving migalastat, a treatment for a rare disorder called Fabry disease. Today, Amicus says that the FDA is reversing the decision, and that there is a clear path to approval. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 11, 2017 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:FOLD Source Type: news

British drugs body to challenge new cost rules in court
LONDON (Reuters) - Britain's pharmaceuticals trade body plans to go to court to challenge new cost rules for medicines for very rare diseases, describing the measures on Monday as "inappropriate and unworkable". (Source: Reuters: Health)
Source: Reuters: Health - July 10, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

The Science of Caregiving: Bringing Voices Together
The National Institute of Nursing Research (NINR) and its partners invite you to a free summit that will provide perspectives across the spectrum of caregiving, including the importance of caregiving across the lifespan as well as current and future directions for research to improve the health of patients and caregivers. The summit will bring together an audience of researchers, advocates, healthcare providers, educators, and others interested in the science of caregiving. Registration is required. Partners: NINR’s partners for this event include the National Cancer Institute, the National Institute on Aging, the Eu...
Source: NN/LM Middle Atlantic Region Blog - July 10, 2017 Category: Databases & Libraries Authors: Hannah Sinemus Tags: Education Health Professionals News from NLM/NIH Source Type: news

Meningitis Vaccine No Protection in Rare Diseases
(MedPage Today) -- Drug treatment for blood condition increases risk of meningococcal disease (Source: MedPage Today Geriatrics)
Source: MedPage Today Geriatrics - July 7, 2017 Category: Geriatrics Source Type: news

New York Hospital Offers to Treat British Baby With Rare Disease
Officials at a leading academic medical center in Manhattan have agreed to admit 11-month-old Charlie Gard as an inpatient, under certain conditions. (Source: NYT Health)
Source: NYT Health - July 6, 2017 Category: Consumer Health News Authors: RONI CARYN RABIN Tags: Gard, Charlie (2016- ) Life-Sustaining Support Systems, Withdrawal Of Hospitals New York-Presbyterian Hospital Source Type: news

NIH and collaborators identify the genomic cause for Carey-Fineman-Ziter syndrome
Rare disease research uncovers new mechanism underlying muscle development. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - July 6, 2017 Category: American Health Source Type: news

Recordati Rare Diseases Introduces New PANHEMATIN(R) (Hemin for Injection) Dosage Strength for Treatment of Acute Intermittent Porphyria (AIP)
Updated Label Reflects More Than 30 Years of Clinical Experience LEBANON, N.J., July 5, 2017 -- (Healthcare Sales & Marketing Network) -- Recordati Rare Diseases Inc., a biopharmaceutical company committed to providing orphan therapies to underserved r... Biopharmaceuticals, Product Launch Recordati Rare Diseases, PANHEMATIN, hemin, Porphyria (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 5, 2017 Category: Pharmaceuticals Source Type: news

Make DNA tests routine, says UK's chief medical officer
Sally Davies calls for making genomic testing as common as blood tests to usher in the era of precision medicine to treat cancers and rare diseasesGenomic testing should become a normal part of NHS care, beginning with cancer patients and those with rare diseases, says the chief medical officer, Dame Sally Davies.In her annual report, Davies stresses her enthusiasm for the genomic revolution which could transform the treatment that NHS patients receive. Drugs can be matched to the disease and to the patient to maximise the benefit and reduce side-effects.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - July 4, 2017 Category: Science Authors: Sarah Boseley Health editor Tags: Health Genetics Cancer research Science NHS Medical research UK news Society Biology Source Type: news

Make DNA tests routine, says England's chief medical officer
Sally Davies calls for making genomic testing as common as blood tests to usher in the era of precision medicine to treat cancers and rare diseasesGenomic testing should become a normal part of NHS care, beginning with cancer patients and those with rare diseases, says the chief medical officer, Dame Sally Davies.Inher annual report, Davies stresses her enthusiasm for the genomic revolution which could transform the treatment that NHS patients receive. Drugs can be matched to the disease and to the patient to maximise the benefit and reduce side-effects.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - July 4, 2017 Category: Science Authors: Sarah Boseley Health editor Tags: Health Genetics Cancer research Science NHS Medical research UK news Society Biology Source Type: news

Chief medical officer calls for gene testing revolution
Offer NHS patients DNA tests to pick the best cancer and rare diseases treatment, England's top doctor says. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 3, 2017 Category: Consumer Health News Source Type: news

Woodlands biopharmaceutical co. to merge with Boston-area startup
Acer Therapeutics, a 3-year-old biotech company developing drugs for ultra-rare diseases, said July 3 that it will go public through a reverse merger with a small Houston-area company. Cambridge, Massachusetts-based Acer said it would merge with The Woodlands-based Opexa Therapeutics (Nasdaq: OPXA). Shares of the combined company — which will retain Acer’s name and Kendall Square headquarters — will be traded under the ticker “ACER.” Opexa laid off all but two of its 20 employees after… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - July 3, 2017 Category: American Health Authors: Max Stendahl Source Type: news