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INNOVATION
11 Feb 2026
Can Nanomedicine Rescue Stalled Rare Drugs?MARKET TRENDS
5 Feb 2026
Rare Disease Drug Boom Sparks Dealmaking and RivalryPARTNERSHIPS
3 Feb 2026
Why Scale Is Suddenly Everything in Rare Disease DrugsMARKET TRENDS
14 Dec 2025
Rare Disease Boom Redefines Pharma Strategy
An Experimental Gene Therapy Signals New Hope for Rare Disorders
INNOVATION
11 Dec 2025
A custom SLC6A1 gene therapy and Mirum’s Bluejay deal point to early, cautious momentum in rare disease innovation
INNOVATION
11 Feb 2026
Can Nanomedicine Rescue Stalled Rare Drugs?
A new nanoparticle platform seeks to solve stubborn formulation hurdles in rare disease drug development
MARKET TRENDS
5 Feb 2026
Rare Disease Drug Boom Sparks Dealmaking and Rivalry
More diagnoses and bold acquisitions are reshaping the orphan drug market, even as pricing and access pressures intensify
PARTNERSHIPS
3 Feb 2026
Why Scale Is Suddenly Everything in Rare Disease Drugs
BioMarin’s Amicus deal highlights a pivot toward scale, steady revenue, and broader portfolios as rare disease companies brace for more me...
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
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