CureDuchenne Biobank has Launched!
CureDuchenne has launched the CureDuchenne Biobank, a neutral, centralized and self-funded data hub that will unlock information to accelerate research. It will provide a consistent set of resources for scientific…
source: pixabay.com
Treatment for Duchenne Muscular Dystrophy with Rare Mutations Accepted Under Accelerated Approval
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…
source: pixabay.com
A New Pediatric Precision Medicine Center is Opening in Utah
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
Progress is Being Made in Duchenne Muscular Dystrophy Gene Therapy
Researchers at the University of Pennsylvania are working on a new potential gene therapy for Duchenne muscular dystrophy (DMD). DMD is a rare form of muscular dystrophy that primarily affects…
source: pixabay.com
Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…
<a href="https://pixabay.com/users/luvqs/">luvqs</a> / Pixabay
Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy
According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
A Middle Schooler with Duchenne Muscular Dystrophy Scores in Football Game
According to a story from 10tv.com, Kelly Maynard could be hear cheering loudly from the sidelines as her son Jackson, aged 12, scored a touchdown in one of his middle…
.jpg)
FDA Approval Was Granted For a Duchenne Muscular Dystrophy Drug Amid Internal Disagreements
The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…
<a href="https://pixabay.com/users/PublicDomainPictures/">PublicDomainPictures</a> / Pixabay
Steadman Philippon Research Institute Receives Award for Duchenne Muscular Dystrophy Research
According to a story from Financial Buzz, the Steadman Philippon Research Institute (SPRI) recently received an award in research from the National Institutes of Health (NIH). The award is in…
<a href="https://pixabay.com/users/rawpixel/">rawpixel</a> / Pixabay
Advocates for Duchenne Muscular Dystrophy Start Company Designed to Collect Data Directly From Patients
According to a story from Xconomy, the US Food and Drug Administration (FDA) made a controversial decision three years ago when it approved the drug eteplirsen as a treatment for…
source: pixabay.com
First Patient Dosed in Phase 3 Study of Experimental Idiopathic Pulmonary Fibrosis Drug
According to a press release from the San Francisco-based FibroGen, Inc., the first patient has been dosed in the Company's phase 3 clinical study of the experimental drug pamrevlumab for…
.png)
The Race is on Between Gene Editing and Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common, yet severe, form of muscular dystrophy, a group of rare neuromuscular disorders. DMD is caused by mutations in the gene that codes…
source: pixabay.com
Early Data Released From Phase 1B Duchenne Muscular Dystrophy Gene Therapy Trial
According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…
<a href="https://pixabay.com/users/3dman_eu/">3dman_eu</a> / Pixabay
Duchenne Muscular Dystrophy and The Heart: Five Facts You Should Know
According to a story from blog.cincinnatichildrens.org, a diagnosis of Duchenne muscular dystrophy is always an alarming piece of news; learning that your child has a progressive muscle atrophy disorder that…
<a href="https://pixabay.com/users/hirskoff/">hirskoff</a> / Pixabay
Community Helps Support Ten Year Old Affected by Duchenne Muscular Dystrophy
According to a story from westernmassnews.com, the community of Monson, Massachusetts is coming together to support Jason Harris and his family. Jason is ten years old and lives with Duchenne…
<a href="https://pixabay.com/users/Capri23auto/">Capri23auto</a> / Pixabay
Company Seeks Marketing Approval for Possible Duchenne Muscular Dystrophy Drug in EU
According to a story from BioPortfolio, the specialty pharmaceutical company Santhera Pharmaceuticals recently announced that it has submitted its marketing application to the European Medicines Agency (EMA) for its drug…
.jpg)
CRISPR-Cas9: Scientists and Patients Have Philosophical Differences
Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries
The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…
The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
<a href="https://pixabay.com/users/Parentingupstream/">Parentingupstream</a> / Pixabay
After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
<a href="https://pixabay.com/users/Parentingupstream/">Parentingupstream</a> / Pixabay
After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
SomaLogic is Working to Improve Precision Medicine for Rare Diseases
The Growth of Precision Medicine Precision medicine is the idea that each patient is an individual, and therefore deserves an individualized treatment plan to secure the best outcomes. It is…
<a href="https://pixabay.com/users/HarinathR/">HarinathR</a> / Pixabay
Duchenne Muscular Dystrophy Patient That Admired Stephen Hawking Passed Away During Exams…With Close to Perfect Scores
According to a story from m.dailyhunt.in, Vinayak Sreedhar, an Indian student with Duchenne muscular dystrophy, passed away in the midst of taking his exams. Vinayak was a driven and highly…
.jpg)
Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance
In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…
