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

Del-zota is designed for patients with mutations amenable to exon 44 skipping, which is just 7% of the overall DMD patient ...

The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

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.

In its complete response letter, the FDA cited insufficient evidence establish deramiocel's effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER ...

The FDA has launched a formal investigation into two reports of fatal acute liver failure in patients treated with Sarepta and Roche's Duchenne muscular dystrophy (DMD) gene therapy Elevidys. The ...

Taiho Pharmaceutical’s investigational Duchenne muscular dystrophy (DMD) therapy, pizuglanstat, has shown no benefit in a Phase III trial.

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

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

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

The U.S. Food and Drug Administration has declined to approve Capricor Therapeutics' cell therapy for a heart condition and ...

ICER’s upcoming report could shed light on how the launch price of newly approved drugs affects overall cost and access from ...