Rare Diseases News
🌏 NMPANMPA breaking news and regulatory updates for BD, investors, and analysts
Showing 1–12 of 12 articles
PMDA Regulatory Updates: New Orphan Drug Portal and ASEAN Symposium Open
Ironwood Pharmaceuticals Survey Reveals Critical Gaps in Short Bowel Syndrome TPN Treatment at DDW 2026
Arrowhead Pharmaceuticals Receives TGA Approval for REDEMPLO (Plozasiran) in Australia for Rare Genetic Disorder
FDA Approves Travere's FILSPARI for FSGS Treatment in Adults and Children 8+
Argo Biopharma Doses First Patient in Phase I Trial of siRNA Therapy BW-50218 for TTR-Related Disease
BioMarin Completes $3 Billion Acquisition of Amicus Therapeutics, Expands Rare Disease Portfolio with Galafold and Pompe Therapies
Spinogenix Launches Phase 2b/3 CLARITY Trial of SPG601 for Fragile X Syndrome Treatment
Argo Biopharma Begins Phase II Trials of BW-40202 siRNA Therapy for Rare Kidney Diseases
PMDA SAKIGAKE Designation: Accelerating Regenerative Medicine Market Access in Japan
NMPA Priority Review Pathway: Accelerating Oncology & Rare Disease Drug Approvals
NMPA Accelerated Approval Orphan Drugs: Impact & Future Trends in China
Pompe Disease Treatments APAC: Market Growth and Regulatory Insights