Duchenne Muscular Dystrophy

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of this year.

Six takeaways from a recent review about the brain-related comorbidities of Duchenne muscular dystrophy

The company says it plans to file for accelerated approval later this year.

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.

The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other diseases, such as systematic sclerosis.

Duvyzat was approved in March 2024 and works to reduce the inflammation and loss of muscle experienced by patients with Duchenne muscular dystrophy.

A number of physicians experienced in treating DMD across the nation contributed to this article recently published in the Annals of the Child of Neurology Society.

Full approval was granted for the one-time gene therapy to ambulatory patients aged 4 years older. The FDA also granted accelerated approval to Elevidys for non-ambulatory patients.

New White Paper looks at the solutions and policy options of how to pay for high-cost gene therapies when there is still so much uncertainty about the outcomes and the durability of clinical benefits.