FDA approves first gene therapy for Duchenne muscular dystrophy
The Food and Drug Administration June 22 approved the first gene therapy to treat patients aged 4-5 with a confirmed mutation in the Duchenne muscular dystrophy gene. As part of the drug’s accelerated approval, the drugmaker must conduct additional clinical trials to verify the predicted clinical benefit.
Related News Articles
Headline
Mounting pressures on the health care workforce have created a crisis with short-term staffing shortages and a long-range picture of an unfulfilled talent…
Headline
Cardiovascular conditions are one of the most common causes of morbidity and mortality among pregnant women. AHA’s Better Health for Mothers and Babies…
Headline
Kittitas Valley Healthcare in rural Washington state last year implemented an innovative new model for retaining essential obstetric and other women’s health…
Blog
Since 2018, Black Maternal Health Week has been a national observance from April 11–17. This annual observance was created by the Black Mamas Matter Alliance…
Headline
Two caregivers discuss how Colorado’s San Luis Valley Hospital creatively maximizes its resources to continue to deliver obstetric services to the families and…
Headline
As part of a yearlong series devoted to rural hospitals and health systems in America, two experts from Intermountain Health discuss their "First 1,000 Days of…