New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Neuromuscular diseases encompass a wide range of disorders characterized by progressive muscle weakness and dysfunction, resulting from abnormalities in the ...

Biogen enhances its portfolio and margins amidst market challenges and competition. Click here to find out why BIIB stock is ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Regenxbio is ranked Strong Buy due to low EV, strong partnerships, royalty income, bullish trends, and buyout potential.

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Scholar Rock (SRRK) announced that the U.S. Food and Drug Administration FDA has accepted its Biologics License Application BLA for ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

We need a different way to pay for these “miracle” drugs. One option is to have them paid for on an annual basis, spreading the cost over the patient’s estimated lifespan, with payment to stop if the ...