New FDA-Approved C3 Glomerulopathy (C3G) Treatment Targets Underlying Cause of Disease
Carla Nester, MD, discusses the recent U.S. FDA approval of iptacopan as the first and only treatment of adults with C3 glomerulopathy (C3G).
Carla Nester, MD, discusses the recent U.S. FDA approval of iptacopan as the first and only treatment of adults with C3 glomerulopathy (C3G).
Arezou Khosroshahi, MD, discusses the approval of Uplizna (inebilizumab-cdon) for treatment of immunoglobulin G4-related disease (IgG4-RD).
The U.S. FDA has approved belzutifan for patients with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma.
Neuroblastoma is a rare childhood cancer characterized by a neuroendocrine tumor originating in neuroblasts or neural crest progenitor cells.
Norman Putzki, MD, Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for SMA.
Dr. Sonal Kumar, discusses the integral role of the patient voice in navigating disease progression and determining an individualized care plan for people living with…
Gavin Lindberg details his family’s experience with neuroblastoma, provides advice to families, and discusses the EVAN Foundation.
Tenosynovial giant cell tumor (TGCT) is a rare benign tumor involving the joint synovium, bursae, and tendon sheath.
Daniel DeFabio, Global Genes and rare disease father, discusses Global Genes’ patient and advocate initiatives.
Sonia Gobeil, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS).
Jean Elwing discusses PAH research leading to better management of people with Pulmonary Arterial Hypertension