Top News

Illustration of mitochondria linked to rare disease drug research and biotech innovation

RESEARCH

20 Sep 2025

From Niche to Notable: Rare Diseases Reshape Biotech

Approval of elamipretide and Mirum Pharma deal point to growing investor interest in high-need therapies with small patient populations

Newborn baby with hearing test and forehead monitoring equipment in a clinical setting

TECHNOLOGY

13 May 2026

Born Deaf No More: FDA Greenlights Historic Treatment

BridgeBio Attruby medicine box for transthyretin amyloid cardiomyopathy

INSIGHTS

30 Oct 2025

BridgeBio’s Strategy Shift Tests the Economics of Rare Disease

Latest News

Gene Therapy Gets a Faster Lane at the FDA

INNOVATION

12 Jun 2026

FDA's new 'plausible mechanism' framework could accelerate gene-editing approvals for ultra-rare diseases with as few as 5 patients

RNA strand illustration representing personalised antisense therapy development

INNOVATION

1 Feb 2025

Inside the Push to Personalize RNA Medicine

Tailored antisense drugs move from concept to clinic as researchers and companies test faster models for ultra-rare conditions

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