Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease ...

Discover six innovative clinical-stage biotech companies fostering R&D in the Duchenne muscular dystrophy therapeutic space.

Givinostat, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed for the treatment of DMD and BMD.

Biotech reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular ...

In a big homecoming night win, Hilliard Davidson's biggest play may have been a Jackson Maynard touchdown that didn't show up ...

A dispute over a Duchenne gene therapy highlights thorny issues surrounding FDA approvals and insurance policies.

In 2023, two potential FDA approvals and three late-stage clinical trial readouts could shift the treatment landscape for Duchenne muscular dystrophy. The upcoming FDA approval decision for ...

A ‘Relive the 80s’ theme marked the occasion, which brought together 740 members of the commercial property industry at the ...

Pratteln, Switzerland, September 24, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that Swissmedic, the Swiss Agency for Therapeutic Products, has accepted for review the marketing ...

In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

The Delhi High Court has directed the central government to establish a national fund for rare diseases, ensuring effective treatment and monitoring for patients suffering from conditions like ...