FORWARD-53 Clinical Trial in Duchenne Muscular Dystrophy Shows Promise
Paul Bolno, MD, discusses results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with DMD amenable to exon 53 skipping.
Related Articles
Newly Approved Treatment Targets Underlying Cause of IgG4-Related Disease
Arezou Khosroshahi, MD, discusses the approval of Uplizna (inebilizumab-cdon) for treatment of immunoglobulin G4-related disease (IgG4-RD).
FDA Approves Treatment for Pheochromocytoma or Paraganglioma
The U.S. FDA has approved belzutifan for patients with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma.
Neuroblastoma
Neuroblastoma is a rare childhood cancer characterized by a neuroendocrine tumor originating in neuroblasts or neural crest progenitor cells.
Positive Safety and Efficacy Data for Intrathecal Administration of Gene Therapy for SMA
Norman Putzki, MD, Novartis, discusses positive safety and efficacy data for OAV101 IT, an investigational gene therapy for SMA.
Navigating Diagnosis and Treatment for Primary Biliary Cholangitis: The Integral Role of the Patient Voice
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…
Neuroblastoma: Evan Foundation
Gavin Lindberg details his family’s experience with neuroblastoma, provides advice to families, and discusses the EVAN Foundation.
Tenosynovial Giant Cell Tumor (TGCT)
Tenosynovial giant cell tumor (TGCT) is a rare benign tumor involving the joint synovium, bursae, and tendon sheath.
Global Genes’ Patient and Advocate Initiatives
Daniel DeFabio, Global Genes and rare disease father, discusses Global Genes’ patient and advocate initiatives.
Ongoing Research for Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Sonia Gobeil, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS).
PAH Clinical Research Highlights: CHEST 2024
Jean Elwing discusses PAH research leading to better management of people with Pulmonary Arterial Hypertension