There are celebrations galore as Jack Johnson – the inspiration for Wigan-based charity Joining Jack – marks his 18th ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates to the BLA in ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

A PepGen drug in development for myotonic dystrophy type 1 (DM1) has encouraging early human data that suggest it could be a better treatment than other medicines in clinical testing for this rare, ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

US FDA grants Orphan Drug Designation to Atossa Therapeutics’ (Z)-endoxifen to treat Duchenne muscular dystrophy: Seattle Monday, January 19, 2026, 17:00 Hrs [IST] Atossa Therap ...