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Rare Diseases
MDA 2026 Spotlights Brogidirsen`s 4.5-Year DMD Data
09 Mar 2026 //
PR NEWSWIRE
Brogidirsen 3.5Y Trial Shows Results in Duchenne MD at WMS 2025
14 Oct 2025 //
PR NEWSWIRE
Buloxibutid Granted Orphan Drug Status In Japan For IPF
30 Sep 2025 //
ACCESSWIRE
FDA Fast Tracks NS-229 for Eosinophilic Granulomatosis Treatment
09 Sep 2025 //
PR NEWSWIRE
NS Pharma, Boston Children`s Hospital Ally for Rare Diseases
01 Jul 2025 //
PR NEWSWIRE
FDA Accepts License Application for MPS II Treatment
14 May 2025 //
PR NEWSWIRE
FDA Grants Orphan Drug Designation to NS-229
21 Apr 2025 //
PR NEWSWIRE
NS-050/NCNP-03 Gets FDA Rare Pediatric Disease Designation
10 Sep 2024 //
PR NEWSWIRE
The Menarini Group Announces ELZONRIS Designated as an Orphan Drug for BPDCN
30 Aug 2023 //
BUSINESSWIRE
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