エピソード

  • 221: An Interview With Dutch Neurologist Ewout Groen of SMA Europe
    2026/04/20

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Dutch neurologist Ewout Groen, PhD, a member of the Scientific Advisory Board of SMA Europe.

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    9 分
  • 220: An Interview With Dr. Jaime Moore on Obesity Medications and Neuromuscular Disease
    2026/04/13

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jaime Moore, MD, about weight loss drugs—specifically the role of GLP-1 receptor agonists in treating children with neuromuscular diseases and obesity.

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    14 分
  • 219: An Interview With Dr. Natalie Truba on the Psychological Aspects of Gene Therapy
    2026/04/07

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric psychologist Natalie Truba, PhD, on the psychological aspects of gene therapy in neuromuscular disease.

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    15 分
  • 218: An Interview With Donna Shipp on Her IgG4-RD Patient Journey
    2026/03/31

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donna Shipp, a Boston-area nurse who was misdiagnosed with cancer, underwent surgery and later realized she had IgG4-RD. She now advocates on behalf of others with this rare disease.

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    14 分
  • 217: An Interview With Abby Bronson of Edgewise Therapeutics About Becker Muscular Dystrophy Awareness
    2026/03/30

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Abby Bronson, vice president of patient advocacy at Edgewise Therapeutics, about a new effort to raise awareness of Becker muscular dystrophy as a disease distinct from Duchenne muscular dystrophy.

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    12 分
  • 216: An Interview With Allison Moore, Founder and CEO of the Hereditary Neuropathy Foundation
    2026/03/23

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. Moore was the winner of the Muscular Dystrophy Association's 2026 Donavon Decker Legacy Award for Community Impact in Research.

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    10 分
  • 215: An Interview With John Crowley, President and CEO of the Biotechnology Innovation Organization
    2026/03/17

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews John Crowley, president and CEO of the Biotechnology Innovation Organization (BIO), founder of Amicus Therapeutics, and father of 2 children with Pompe disease.

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    14 分
  • 214: An Interview With Dr. Hyun Kim, Director of the University of Minnesota's Interstitial Lung Disease Program
    2026/03/02

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Hyun Kim, MD, professor of medicine and director of the University of Minnesota's Interstitial Lung Disease Program, about idiopathic pulmonary fibrosis.

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    13 分