BridgeBio Pharma Inc. BBIO on Wednesday announced a recent presentation of interim results from its Phase 3 FORTIFY trial for BBP-418, a treatment for limb-girdle muscular dystrophy type 2I/R9.

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

MRI results highlighted that patients with baseline cardiomyopathy saw a 3.3 percentage-point improvement in left ventricular ejection fraction. ・Capricor has already submitted a BLA for Deramiocel to ...

– 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 ...

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 ...

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 ...

Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.

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 ...

Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...