Capricor Therapeutics has publicly released its July response to the FDA's rejection of its Duchenne muscular dystrophy cell therapy deramiocel after the agency included deramiocel in a new batch of ...

Leerink analyst Joseph Schwartz raised the firm’s price target on Sarepta (SRPT) to $15 from $12 and keeps a Market Perform rating on the shares.

Drug companies that specialize in care for rare diseases are growing frustrated with the FDA, despite agency leadership’s ...

Amid an unprecedented turnover in leadership at the FDA and mass layoffs of staff, communication has crumbled and uncertainty ...

Exclusive: Alison Joseph's sons Noah and Hunter both have Duchenne muscular dystrophy. She tells Rhian Lubin the ...

These once high-flying stocks no longer seem to have promising prospects. Just because a stock has been battered doesn't make ...

There is no cure for muscular dystrophy, (MD) but the right treatment approach can ease symptoms for people with these rare genetic disorders.

Japanese drugmaker Chugai announced that it has implemented measures based on safety evaluations for Elevidys intravenous infusion (delandistrogene moxeparvovec), a regenerative medical product for ...

Long before there was Sarepta's gene therapy for muscular dystrophy, there was Donavon Decker and his offer to test a gene ...

Chugai Pharma implemented measures based on safety evaluations for Elevidys Intravenous Infusion: Tokyo Friday, September 5, 2025, 12:00 Hrs [IST] Chugai Pharmaceutical Co., Ltd.

Despite decades of research and the approval of several therapies to help manage symptoms, there is still no cure for ...

As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable ...