After Driving An N-of-1 Therapy For Her Son, An Advocate Turns To Helping Others RARECast podcast

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After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others

3M ago 30:48

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Innehåll tillhandahållet av RARECast. Allt poddinnehåll inklusive avsnitt, grafik och podcastbeskrivningar laddas upp och tillhandahålls direkt av RARECast eller deras podcastplattformspartner. Om du tror att någon använder ditt upphovsrättsskyddade verk utan din tillåtelse kan du följa processen som beskrivs här https://sv.player.fm/legal.

When Yiwei She’s son Leo was diagnosed with a severe neurodevelopmental condition, he was one of only two people known to have the ultra-rare disease. In a year, though, working with Creyon Bio, the family was able to move from the start of research for an ASO to treating Leo with the experimental therapy. We spoke to She, founder of the TNPO2 Foundation, about how her family was able to treat her son Leo with an experimental ASO with relative speed, the work the TNPO2 Foundation is doing to accelerate the diagnosis of other children with ultra-rare conditions, and its efforts to find accessible and affordable pathways to treatments for others.

… continue reading

521 episoder

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

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After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others

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published 3M ago

Dela

MP3•Episod hem

Innehåll tillhandahållet av RARECast. Allt poddinnehåll inklusive avsnitt, grafik och podcastbeskrivningar laddas upp och tillhandahålls direkt av RARECast eller deras podcastplattformspartner. Om du tror att någon använder ditt upphovsrättsskyddade verk utan din tillåtelse kan du följa processen som beskrivs här https://sv.player.fm/legal.

When Yiwei She’s son Leo was diagnosed with a severe neurodevelopmental condition, he was one of only two people known to have the ultra-rare disease. In a year, though, working with Creyon Bio, the family was able to move from the start of research for an ASO to treating Leo with the experimental therapy. We spoke to She, founder of the TNPO2 Foundation, about how her family was able to treat her son Leo with an experimental ASO with relative speed, the work the TNPO2 Foundation is doing to accelerate the diagnosis of other children with ultra-rare conditions, and its efforts to find accessible and affordable pathways to treatments for others.

… continue reading

521 episoder

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

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