hotspot -WSS

hotspot

author：hotspot 2025-09-27 12:47:34 - browse：518

Duchenne muscular dystrophy is a devastating disease and, until very recently, was one without much hope. When Hawken Miller was diagnosed at age 5, the physician told his parents to enjoy the time they had with him, as there wouldn’t be much. Over 20 years later, Miller is a journalist and content strategist for CureDuchenne, an organization started by his mother when he was diagnosed, and new treatment is on the horizon.

Last month, the FDA approved a new gene therapy from Sarepta Therapeutics that will provide hope for many patients and families. The new drug, however, has its limitations — it’s only approved for children age 4-5, for example. Miller joined the podcast to discuss how this drug may change lives, what more needs to happen, and what his personal experience has been living with Duchenne.

“For the older boys, we’re running up against the clock a little bit with what we’re able to do and what we’re not able to do,” Miller said. “And if we’re able to try anything that could possibly help, I think most people would like the opportunity to do that.”

The conversation is based off his recent First Opinion, “The FDA’s approval of a new gene therapy for Duchenne muscular dystrophy won’t help me — but it gives me hope.”

Be sure to sign up for the weekly “First Opinion Podcast” on Apple Podcasts, Stitcher, Google Play, or wherever you get your podcasts.

Previous article： Jerome Adams on the need for new antivirals for Covid
Next article： How the political right came to back psychedelics to treat trauma

Wikipedia

WSS

Iowa House, Senate pass 6

A virus, fished out of a lake, may have saved a man’s life

DOJ reversal could clear way for Carroll's 1st lawsuit against Trump to proceed

Elizabeth Holmes on track for early release from her 11

The future of health care in the House

Biden launches a new push to limit health care costs

hotspot

hotspot

explore

Psychedelics group wrestles with new pharma identity

AI can decode, harness the power of the humane immune system

Medicare proposal to cover medical devices draws a mixed reaction

Threats against the LGBTQIA+ community intensifying: Department of Homeland Security

The cancer drug shortage isn’t new — and neither are the solutions

Insurance companies sue energy corporation after it was blamed for helping start Colorado wildfire

In Memoriam: Notable people who died in 2023

Why 40% Americans with Parkinson’s don’t see a neurologist

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Threats against the LGBTQIA+ community intensifying: Department of Homeland Security

knowledge

Why Charlie Gard's case is so disturbing to Americans

Beach reads and more: STAT's 2023 summer book list

Why Charlie Gard's case is so disturbing to Americans

Chemotherapy drug shortage eases slightly but still widespread

California’s proposed $25 per hour health care minimum wage

FDA authorizes Novavax’s updated Covid

Wikipedia

Wikipedia

focus

focus