by James Radke, PhD and Madaline Spencer | Jun 14, 2023
Dean Suhr, of the MLD Foundation, provides an overview of the philosophy of their foundation when it comes to supporting research for metachromatic leukodystrophy (MLD). MLD is a genetic, lysosomal disorder caused by a deficiency in the enzyme arylsulfatase A...
by Madaline Spencer and James Radke, PhD | Jun 13, 2023
Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, discusses clinical trial development challenges for ultra-rare diseases. Stealth Biotherapeutics is developing elamipretide, a mitochondrial protective agent, as a possible treatment...
by Madaline Spencer and James Radke, PhD | Jun 13, 2023
Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains current treatment options for patients with primary Immunodeficiency (PI). PI is a heterogeneous group of inherited disorders characterized by impaired immune...
by Madaline Spencer and James Radke, PhD | Jun 12, 2023
Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains why it often takes years to properly diagnose Primary Immunodeficiency (PI). PI is a heterogeneous group of inherited disorders characterized by impaired immune...
by Madaline Spencer | May 31, 2023
Sitra Tauscher-Wisniewski, MD, Vice President of Clinical Development & Analytics at Novartis Gene Therapies, discusses long-term data for onasemnogene abeparvovec (Zolgensma), an FDA-approved gene therapy for spinal muscular atrophy (SMA). SMA is a...
