Dyne Therapeutics, Inc. develops treatments for muscle diseases using its FORCE platform, with promising candidates DYNE-101 ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is ...

The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

Columnist Shalom Lim shares how his business, Rebirth Ensemble, will allow him to pursue visual arts and support artists with disabilities.

Regulating drug discovery and pharmaceutical manufacturing, the FDA ensures the safety and effectiveness of innovative ...

Duchenne, has been a transformative force in the fight against Duchenne muscular dystrophy (DMD). Following her son Hawken’s ...

International Assets Investment Management LLC increased its stake in Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Free Report) by 59,222.7% in the 3rd quarter, Holdings Channel.com reports. The fund ...

The researchers will explore non-viral options for delivering gene therapy to potential DMD patients, specifically ...