by Madaline Spencer | May 16, 2025
The U.S. Food and Drug Administration (FDA) approved Welireg (belzutifan) for patients ages 12 years and older with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma. Pheochromocytoma and paraganglioma are rare tumors that develop from...
by Madaline Spencer | May 16, 2025
Al Freedman, PhD, Rare Disease Psychologist and Rare Dad, discusses how industry partners can support the mental health of rare disease communities. Dr. Freedman provides individual counseling, support group facilitation, and advises rare disease...
by Madaline Spencer | May 15, 2025
Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, provides an overview of urea cycle disorders (UCDs) and discusses developing novel therapies. UCDs are a group of rare, inherited metabolic disorders caused by deficiency of one of the six...
by Madaline Spencer | May 14, 2025
Arezou Khosroshahi, MD, Associate Professor of Medicine at Emory University School of Medicine, discusses the approval of Uplizna (inebilizumab-cdon) for treatment of immunoglobulin G4-related disease (IgG4-RD). IgG4-RD is an immune-mediated condition...
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)...
