While Sarepta’s gene therapy for the treatment of DMD called Elevidys was approved by the FDA in 2023, Regenxbio is ...

Biotech company Regenxbio and the University of Pennsylvania on Friday convinced a U.S. appeals court to revive their patent lawsuit against Sarepta Therapeutics over Sarepta's Duchenne muscular ...

Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...

– Company to host an Industry Forum to discuss the lived experience of Becker and clinical advancements featuring a leading neuromuscular disease expert and a patient advocate – Edgewise is sponsoring ...

Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...

Please provide your email address to receive an email when new articles are posted on . New therapies for muscular dystrophy must go beyond traditional corticosteroid administration. Treatments that ...

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive ...

Minnesota (WCCO) -- An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment. Colton Belluzzo was diagnosed with a form of muscular ...

A research team led by Associate Professor Hidetoshi Sakurai and Researcher Nana Takenaka-Ninagawa recently demonstrated the superior therapeutic potential of iPS cell-derived mesenchymal stromal ...

PTC Therapeutics withdrew a New Drug Application for Translarna, a muscular dystrophy treatment, after the Food and Drug Administration indicated it would likely not grant approval.