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4 News Found

EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome
Drug Approval | January 23, 2024

EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome

GC1130A has previously achieved notable milestones by securing both RPDD and ODD from the U.S. FDA in January 2023


Sanofi reaffirms commitment to rare diseases in India
News | February 28, 2023

Sanofi reaffirms commitment to rare diseases in India

Receives Subject Expert Committee recommendation to bring new therapies of Pompe, ASMD to India


Takeda to collaborate with JCR Pharma to commercialise next-gen Hunter Syndrome therapy
Biotech | October 01, 2021

Takeda to collaborate with JCR Pharma to commercialise next-gen Hunter Syndrome therapy

Takeda to exclusively commercialize JR-141 outside the U.S. (except Japan and certain other Asia-Pacific countries) upon regulatory approval