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 Officer of ...
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 prophylaxis ...
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
Last of the Seven Summits: Making History for Hemophilia!
Mountaineer Chris Bombardier is set to scale the Last of the Seven Summits, Mt. Vinson, becoming the first person with hemophilia 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(document.getElementBy...
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’d freeze,...
Source: cancerslayerblog - November 26, 2017 Category: Cancer & Oncology Tags: MFA writing/speaking 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’d freeze,...
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 pro...
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 breakfast is ready, I en...
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
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Cavity Sam is the character on the board...
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: Wu L...
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 companyR...
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 it. ...
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 affects few...
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
