Clinical

Articles

Interim analysis compared ataluren in a real-world setting from the STRIDE registry with a matched cohort; the final data from the STRIDE registry are expected in 2025.

Ataluren Extends Ability to Walk in Patients With Duchenne Muscular Dystrophy

The profound consequences of the results guide decisions about testing. Who should be tested? And, just as important, who should not?

Genetic Testing for Huntington Disease

Podcasts

In this podcast, Heidi Moawad, MD interviews Cesar Ochoa-Lubinoff, MD, MPH, FAAP, about recent encouraging clinical trial results in patients with Angelman syndrome.

Genetic Disorders

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