by Madaline Spencer and Peter Ciszewski | Jun 15, 2023
Pierantonio Russo, MD, Chief Medical Officer at Eversana, discusses the expanding role artificial intelligence (AI) plays in the diagnostics of rare conditions. As noted by Dr. Russo, diagnostic delays are expected in rare diseases due to many factors....
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...
