127 episodes

Rare Disease Advisor's Rare Care Podcast features exclusive interviews with experts and stakeholders from the rare disease community.

Rare Care Podcast Rare Care Podcast

    • Health & Fitness

Rare Disease Advisor's Rare Care Podcast features exclusive interviews with experts and stakeholders from the rare disease community.

    An Interview with Avner Reshef, MD, Top Israeli Expert in Hereditary Angioedema

    An Interview with Avner Reshef, MD, Top Israeli Expert in Hereditary Angioedema

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Israeli expert Avner Reshef, MD, director of the Allergy, Immunology, and Angioedema Center at Barzilai University Medical Center in Ashkelon, only a few miles from the border with war-torn Gaza.

    • 17 min
    An Interview With Pediatric Neurologist Edward Smith, MD, on the FDA Approval of Vamorolone for Boys With Duchenne Muscular Dystrophy

    An Interview With Pediatric Neurologist Edward Smith, MD, on the FDA Approval of Vamorolone for Boys With Duchenne Muscular Dystrophy

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews pediatric neurologist Edward Smith, MD, about the recent approval of vamorolone, a synthetic corticosteroid, in boys with Duchenne muscular dystrophy.

    • 15 min
    An Interview With Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association

    An Interview With Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Sharon Hesterlee, PhD, chief research officer at the Muscular Dystrophy Association, about the recent proliferation of gene therapies and other treatments for neuromuscular diseases.

    • 7 min
    An Interview With Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, About Living With Spinal Muscular Atrophy

    An Interview With Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, About Living With Spinal Muscular Atrophy

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, at the MDA's 2024 Clinical & Scientific Conference in Orlando, Florida. Walker, 39, has spinal muscular atrophy (SMA) type 2.

    • 12 min
    An Interview With Seema Lalani, MD, Director of Project GIVE, a Pediatric Rare Disease Screening Program in the Rio Grande Valley of Texas

    An Interview With Seema Lalani, MD, Director of Project GIVE, a Pediatric Rare Disease Screening Program in the Rio Grande Valley of Texas

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Seema Lalani, MD, of Baylor College of Medicine in Houston, Texas. Dr. Lalani directs Project GIVE (Genetic Inclusion by Virtual Evaluation), a pediatric screening program in the impoverished 4-county Rio Grande Valley region of southern Texas.

    • 11 min
    An Interview With Matt Trudeau of ITF Therapeutics, on Givinostat as a Therapy for Duchenne Muscular Dystrophy

    An Interview With Matt Trudeau of ITF Therapeutics, on Givinostat as a Therapy for Duchenne Muscular Dystrophy

    Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Matt Trudeau, head of ITF Therapeutics—a division of Italy's Italfarmaco—on treating Duchenne muscular dystrophy with givinostat, a histone deacetylase inhibitor.

    • 9 min

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