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.…
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…
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)
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.…
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…
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…
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)
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…
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…
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…
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…
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)
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…
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 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…
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…
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…
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…
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…
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)
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…
A recent publication from Charcot-Marie-Tooth News announced that this year, for the first time ever, the Muscular Dystrophy Association will be hosting its annual clinical conferences at the same event…
For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF).…
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
