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 […]
Tag: Rare Disease
Zeal Specialty Pharmacy Credentialed in Record Time
Since opening their doors in March of 2023, Zeal Specialty Pharmacy has obtained licensure in all 50 states, Specialty Pharmacy Accreditation from URAC and the Accreditation Commission for Health Care (ACHC), and a Rare Disease & Orphan Drug Designation from ACHC – a feat never accomplished in such a short period of time. By achieving […]
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 […]
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 […]