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MARKET TRENDS
14 Dec 2025
Rare Disease Boom Redefines Pharma StrategyINNOVATION
11 Dec 2025
An Experimental Gene Therapy Signals New Hope for Rare DisordersINVESTMENT
9 Dec 2025
High Risk, High Impact: Rare Disease Biotech Reclaims Investor AttentionTECHNOLOGY
8 Dec 2025
AI Models Take on Medicine’s Rarest Challenges
BioCryst Bets on Convenience in High-Stakes HAE Deal
PARTNERSHIPS
4 Dec 2025
Pending Astria acquisition centres on long-acting therapy as drugmakers seek more convenient treatments for rare diseases
MARKET TRENDS
14 Dec 2025
Rare Disease Boom Redefines Pharma Strategy
Big drugmakers step up investment in niche therapies as science and partnerships turn small patient groups into core growth areas
INNOVATION
11 Dec 2025
An Experimental Gene Therapy Signals New Hope for Rare Disorders
A custom SLC6A1 gene therapy and Mirum’s Bluejay deal point to early, cautious momentum in rare disease innovation
INVESTMENT
9 Dec 2025
High Risk, High Impact: Rare Disease Biotech Reclaims Investor Attention
Mirum’s agreement to buy Bluejay highlights renewed investor interest and growing competition for late-stage rare disease assets
TECHNOLOGY
8 Dec 2025
AI Models Take on Medicine’s Rarest Challenges
AI is speeding up rare disease research and reshaping drug discovery worldwide
PARTNERSHIPS
4 Dec 2025
BioCryst Bets on Convenience in High-Stakes HAE Deal
Pending Astria acquisition centres on long-acting therapy as drugmakers seek more convenient treatments for rare diseases
REGULATORY
17 Nov 2025
FDA Floats Faster Route for Ultra-Rare Therapies
A new FDA proposal could speed rare disease drug approvals using small trials, with tighter science standards and long-term patient follow...
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