explore -WSS

explore

author：fashion 2025-09-30 18:54:38 - browse：562

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： State Dept. review finds Biden bears some blame for Afghanistan failures
Next article： TytoCare raises $49 million to build out AI

leisure time

WSS

Midwives could help bridge maternal health disparities in the U.S.

What the cancer research community can learn from the Covid response

Nearly two

FDA panel to weigh safety of artificial wombs for preterm births

OTC birth control: A brief history of over the counter pill design

FDA panel to weigh safety of artificial wombs for preterm births

hotspot

explore

focus

ChatGPT in medicine: STAT answers readers' burning questions

Patients, doctors press FTC to beef up hospital merger scrutiny

Guidelines to prevent youth baseball injuries need more muscle

Akili changes sales model for its ADHD video game treatment

What does generative AI mean for health care? We asked experts

Transgender sports debate: Physicians say bans are health issue

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

What Mike Pence has said that could be used in a Donald Trump trial

Guidelines to prevent youth baseball injuries need more muscle

For BCIs, commercial hurdles may be bigger than scientific ones

leisure time

Transgender sports debate: Physicians say bans are health issue

OTC birth control: A brief history of over the counter pill design

FDA weighs limits of animal data in assessing artificial wombs

Attacks on the drug industry will lead to fewer breakthroughs

Grad students accuse University of California of wage theft

White House drug shortage group privately met with industry

explore

leisure time

hotspot

explore