Capricor Therapeutics (CAPR) on Friday said the U.S. Food and Drug Administration (FDA) has refused to approve its lead cell ...

In its complete response letter, the FDA cited insufficient evidence establish deramiocel's effectiveness for cardiomyopathy ...

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

Second death linked to Sarepta Therapeutics treatment leaves Duchenne muscular dystrophy community reeling, ... His own 10-year-old son, Konner, received the treatment, called Elevidys, ...

Taiho Pharmaceutical has suffered a blow to its near-term pipeline after a drug candidate for Duchenne muscular dystrophy ...

The company said it was optimistic that the issues identified by the Food and Drug Administration could be resolved with new data.

TAS-205 showed no significant impact on motor function in patients with Duchenne muscular dystrophy (DMD), highlighting the ongoing search for effective treatments for the rare condition.

Duchenne Muscular Dystrophy Market Size, Epidemiology, In-Market Drugs Sales, Pipeline Therapies, and Regional Outlook ...

Sarepta is facing a shareholder lawsuit over ELEVIDYS-related deaths. Robbins LLP cites safety risks and stock losses.

The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR ...

About Robbins LLP : A recognized leader in shareholder rights litigation, the attorneys and staff of Robbins LL have been dedicated to helping shareholders recover losses, improve corporate governance ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...