Recent Videos
Social Wall
Repurposing Shelved Assets for Rare Diseases
Ethical Concerns in Rare Diseases and Expanded Access Programs
The Diversity of Patient Advocacy Group Initiatives
CAMP4 Therapeutics’ Programs For Urea Cycle Disorders and SYNGAP1
A Patient’s Diagnostic Journey With Idiopathic Pulmonary Hemosiderosis (IPH)
Navigating the Challenges of the Orphan Drug Market
Newly Approved Treatment Targets Underlying Cause of IgG4-Related Disease
Mental Health and the Rare Disease Community
Urea Cycle Disorders: Overview and Developing Novel Therapies
Positive Safety and Efficacy Data for Intrathecal Administration of Gene Therapy for SMA
Challenges Faced by Rare Disease Patients in India
Global Genes’ Patient and Advocate Initiatives
Ongoing Research for Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Progressive Familial Intrahepatic Cholestasis: Diagnosing, Treating, Monitoring
Pathophysiology of PFIC
Repurposing Shelved Assets for Rare Diseases
CheckRare May 15, 2025 11:20 am