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author：hotspot 2025-09-29 13:31:03 - browse：142

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.

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