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81: One Duchenne patient's bittersweet hope for new treatment
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Manage episode 371682781 series 2969153
Sisällön tarjoaa First Opinion Podcast. First Opinion Podcast tai sen podcast-alustan kumppani lataa ja toimittaa kaiken podcast-sisällön, mukaan lukien jaksot, grafiikat ja podcast-kuvaukset. Jos uskot jonkun käyttävän tekijänoikeudella suojattua teostasi ilman lupaasi, voit seurata tässä https://fi.player.fm/legal kuvattua prosessia.
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.
…
continue reading
109 jaksoa
MP3•Jakson koti
Manage episode 371682781 series 2969153
Sisällön tarjoaa First Opinion Podcast. First Opinion Podcast tai sen podcast-alustan kumppani lataa ja toimittaa kaiken podcast-sisällön, mukaan lukien jaksot, grafiikat ja podcast-kuvaukset. Jos uskot jonkun käyttävän tekijänoikeudella suojattua teostasi ilman lupaasi, voit seurata tässä https://fi.player.fm/legal kuvattua prosessia.
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.
…
continue reading
109 jaksoa
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