NS Pharma NS-229: Orphan Drug Designation for EGPA Treatment
Drug Updates

The European Commission Grants Orphan Drug Designation to NS-229 for the Treatment of Eosinophilic Granulomatosis with Polyangiitis

NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd, announced today that the European Commission (EC) has granted orphan drug designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA). The orphan drug designation by the EC is issued for drugs which are intended to […]

AOC 1001 Myotonic Dystrophy Treatment
Drug Updates

Avidity Biosciences Announces New Positive AOC 1001 Data Demonstrating Improvement in Multiple Additional Functional Endpoints and Favorable Long-term Safety and Tolerability in People with Myotonic Dystrophy Type 1

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced new positive AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in people living with myotonic dystrophy type 1 (DM1). AOC 1001, Avidity’s lead clinical […]

Hospital Updates

12 Lakh+ Donors Contributed Over INR 125 Cr for Rare Disease Patients on ImpactGuru: Report

ImpactGuru.com, India’s leading integrated healthcare financing platform, reports one-of-its-kind, rare disease online donation crowdfunding movement with 12,00,000+ donors raising INR 125+ crore through 1200+ rare disease crowdfunding campaigns in 14 months (January 2021 to February 2022), for patients fighting rare diseases like Spinal Muscular Atrophy (SMA type 1 & 2), Gaucher’s disease, Pompe’s disease, rare […]