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An Interview With Pediatric Neurologist Edward Smith, MD, on the FDA Approval of Vamorolone for Boys With Duchenne Muscular Dystrophy
Season 1 Episode 119
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 Duch…
Published on 1 year, 8 months ago
An Interview With Sharon Hesterlee, PhD, Chief Research Officer at the Muscular Dystrophy Association
Season 1 Episode 118
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 th…
Published on 1 year, 8 months ago
An Interview With Ira Walker, the Muscular Dystrophy Association's 2024 National Ambassador, About Living With Spinal Muscular Atrophy
Season 1 Episode 117
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 …
Published on 1 year, 8 months ago
An Interview With Seema Lalani, MD, Director of Project GIVE, a Pediatric Rare Disease Screening Program in the Rio Grande Valley of Texas
Season 1 Episode 116
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 Virtu…
Published on 1 year, 8 months ago
An Interview With Matt Trudeau of ITF Therapeutics, on Givinostat as a Therapy for Duchenne Muscular Dystrophy
Season 1 Episode 115
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 givinosta…
Published on 1 year, 9 months ago
An Interview With Thomas Holm Pedersen, PhD, Cofounder and CEO of Denmark's NMD Pharma
Season 1 Episode 114
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Thomas Holm Pedersen, PhD, cofounder and chief executive of NMD Pharma, a Danish company that's pursuing the use of chloride ch…
Published on 1 year, 9 months ago
An Interview With Courtney Silverthorn, PhD, Associate VP of Science Partnerships at the Foundation for the National Institutes of Health
Season 1 Episode 113
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Courtney Silverthorn, PhD, of the Foundation for the National Institutes of Health (FNIH) about the FNIH's Bespoke Gene Therapy…
Published on 1 year, 9 months ago
An Interview With Cristol Barrett O'Loughlin, CEO of Raregivers
Season 1 Episode 112
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Cristol Barrett O'Loughlin, founder and CEO of Raregivers—a nonprofit organization that offers mental health and wellness servi…
Published on 1 year, 10 months ago
An Interview With Omar Kamlin, MD, Senior Medical Director at Orphalan
Season 1 Episode 111
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Omar Kamlin, MD, senior medical director at French drugmaker Orphalan, whose therapy, Cuvrior, treats Wilson disease.
Published on 1 year, 10 months ago
An Interview With Melinda Bachini, Chief Patient Officer at the Cholangiocarcinoma Foundation
Season 1 Episode 110
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Melinda Bachini, chief patient officer at the Cholangiocarcinoma Foundation, about a new study detailing the burden on caregive…
Published on 1 year, 10 months ago