Rewriting the Rules: How Tiny Molecules Are Unlocking a Giant Future for Medicine
The Unbearable Wait: India's Rare Disease Community Caught in a Policy Labyrinth
The Unfolding Story of Hope: BridgeBio's Double Victory in the Battle Against Rare Disease
Biotech's Pulse Check: AI Rises, a Cancer Test Stumbles, and a Rare Disease Shines
Freya's Fight and the Miracle of Gene Therapy: A New Dawn for MLD Patients
A Beacon of Hope: Gene Therapy Milestone Honors Jesse Den Haan's Legacy in the Fight Against Duchenne Muscular Dystrophy
Solid Biosciences Forges Strategic Alliance with Kinea Bio for Friedreich's Ataxia Gene Therapy, Bolstering Focus on DMD Pipeline
PTC Therapeutics: A New Titan Rising to Challenge BioMarin's Reign in Rare Disease Treatment