Parent Project Muscular Dystrophy (PPMD) has added a pre-conference workshop to this year’s 23rd Annual Connect Conference, inviting international medical experts…
Articles by Margarida Azevedo, MSc
Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused…
Raxone (idebenone) has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular…
Sarepta Therpeutics and Genethon have signed a research collaboration agreement to jointly develop a gene therapy for Duchenne…
On June 20, the Massachusetts-based FSH Society and the FSHD Champions ask everyone to join World FSHD Day to…
Advocates from Save Our Sons Duchenne Foundation (SOS), in partnership with Parent Project Muscular Dystrophy (PPMD), have launched Australia’s…
Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045…
A gene therapy treatment may restore the genetic mutations of myotubular myopathy, a type…
A new clinical-stage biotech, Myonexus Therapeutics, is developing gene therapies for limb-girdle muscular dystrophies (LGMDs). Launch plans include the…
The U.S. Food and Drug Administration (FDA) scheduled a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting with PTC…