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
In this conversation, Kardie Tobb, D.O., a non-invasive preventative cardiologist at Cone Health, explains how multidisciplinary teams are breaking down…
Headline
The Health Resources and Services Administration Aug. 12 announced a modernized framework for addressing national newborn screening topics, which includes…
Headline
The Health Resources and Services Administration’s Rural Maternity and Obstetrics Management Strategies Program has released a report highlighting how three…
Headline
The AHA provided comments July 21 to the Senate Committee on Health, Education, Labor and Pensions on price transparency, nursing workforce and rural…
Headline
The Health Resources and Services Administration Maternal and Child Health Bureau has announced grant opportunities available supporting maternal and child…
Headline
In this conversation, Southwest Health’s Kevin Carr, M.D., family medicine physician, and Melissa Carr, M.D., OB/GYN, reflect on the joy of practicing medicine…