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

Panelists discuss how payers seek good return on investment when evaluating expensive gene therapies, creating potential friction when innovative treatments come at significant costs, requiring ...

Capricor Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA on the BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy patients. The company’s CEO said ...

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

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli ...

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 ...

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

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

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

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

Households incur substantial costs to accommodate Duchenne muscular dystrophy (DMD) patients, according to a survey of U.S.

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