by Madaline Spencer | May 13, 2025
Norman Putzki, MD, Global Development Head of Neuroscience and Gene Therapy at Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for spinal muscular atrophy (SMA). It is similar to Zolgensma (onasemnogene abeparvovec)...
by Madaline Spencer | May 12, 2025
Ramaiah Muthyala, PhD, Research Associate Professor at University of Minnesota and President and CEO of Indian Organization for Rare Diseases (IORD), discusses the challenges faced by rare disease patients in India. As Dr. Muthyala explains, rare disease...
by Madaline Spencer | May 9, 2025
Daniel DeFabio, Director of Community Engagement and Education at Global Genes, Co-Founder of Disorder: The Rare Disease Film Festival, and rare disease father, discusses Global Genes’ patient and advocate initiatives. Global Genes is an organization...
by Madaline Spencer | May 8, 2025
Sonia Gobeil, co-founder of Ataxia of Charlevoix-Saguenay Foundation, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS). ARSACS is a rare neuromuscular disorder characterized by...
by Madaline Spencer | May 5, 2025
Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval. As noted by Mr. Suhr, the recent termination of the Advisory Committee on Heritable...
