Diagnosis of Friedreich Ataxia

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD).

African American and Latino older adults with Alzheimer disease and related dementias and their families are likely to face disproportionately high burdens, primarily associated with unpaid caregiving, suggesting the need for policies that may reduce economic burdens for all US residents.

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.

Explore 5 rare genetic and neurological disorders named after pioneering women in medicine, highlighting their significant contributions to previously unknown conditions.

The FDA cleared marketing for the first in vitro diagnostic device that tests blood to aid in diagnosing Alzheimer disease.

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.