Duchenne Muscular Dystrophy

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.