My name is A. Sabesan. I am 25 years old, living in Chennai, Tamil Nadu, India. Up until the age of 18, my life was completely ordinary. But at 19,…
When I was seven, my parents were trying to understand why I was becoming ill. I spent time in hospitals and underwent painful muscle biopsies. Doctors knew I had a…
“For people living with a progressive rare disease, time is measured differently. Research and clinical trials can take years while patients continue to lose strength and function,” said Kathryn Bryant…
As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has granted priority review to BridgeBio Pharma’s investigational therapy, BBP-418, marking a key regulatory milestone for a condition that…
BridgeBio Pharma has reported decisive success in a pivotal phase 3 trial for its rare disease drug BBP-418, aimed at treating limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). Reported by Fierce…
On July 18, 2025, the U.S. Food and Drug Administration (FDA) took decisive action against Sarepta Therapeutics following the deaths of three individuals who had received the company’s gene therapy…
According to a story from BioPharma Dive, the drug company BridgeBio has been working on the development of a new treatment for limb-girdle muscular dystrophy, a rare disease. At the…
Written by Lacey Woods From a young age, I knew I wanted to be a mother, to have biological children and also to adopt. This desire stayed with me throughout…
Statins are a type of drug that can help you to lower your blood cholesterol levels. These drugs work by inhibiting HMG CoA reductase, an enzyme that the body…
According to a recent article, Dr. Jyoti K. Jaiswal shared his research pertaining to gene therapies to be used to treat limb-girdle muscular dystrophy 2B (LGMD2B). Limb-Girdle Muscular Dystrophy (LGMD)…
In the National Football League's My Cause My Cleats campaign, NFL players are able to show their passions beyond the game and represent a cause that is important to them through customized…
In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…
While gene therapy is a burgeoning technique to treat a number of rare genetic conditions, there are still a few complexities associated with this treatment option. How safe and effective…
According to a story from One News Page, the biopharmaceutical company BridgeBio Pharma and its affiliate ML Bio Solutions have recently announced that dosing has begun in its phase 2…
At the end of September, Sarepta Therapeutics ("Sarepta") announced positive data from a study exploring SRP-9003, a gene therapy candidate, as a potential treatment for patients with limb-girdle muscular…
In a recent press release, biotechnology company Sarepta Therapeutics announced positive findings from their clinical trial studying SRP-9003 as a treatment for limb-girdle muscular dystrophy (LGMD) type 2E. This…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
According to a story from Science Daily, a recent study has found that lithium chloride was able to improve muscle size and strength in a mouse mode of a specific…
A recent study published in the journal Nature Communications highlights a potential therapy for the treatment of muscular dytrophies and other diseases linked to mutations of the FKRP gene. In the study, the…
According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…
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