rare diseases

dominoes stop stall halt end red
Biotech

Genentech drug fails rare disease study, raising obesity questions

Genentech will not push anti-myostatin antibody emugrobart into phase 3 for two muscle-wasting diseases after it failed to improve muscle growth.
Darren Incorvaia Mar 20, 2026 2:11pm
MPS Families Carry Coffin to FDA

Rare disease advocates stage 'funeral' at FDA, make 4 demands

Mar 18, 2026 5:25pm
negative time concept

Stoke aims to restore developmental loss in Dravet syndrome

Mar 18, 2026 2:57pm
halfway there

Ultragenyx gene therapy lessens ammonia levels in ph. 3

Mar 12, 2026 11:33am
Graphic of a line of blocks with percentages on them with one block in yellow showing an arrow increasing

Small molecules are 'resurgent' in orphan drug R&D: Evaluate

Mar 12, 2026 10:30am
Metallic arrow symbols illuminated by blue and magenta lights forming a graph on blue and magenta background

BridgeBio's muscle weakness data impress ahead of FDA filing

Mar 12, 2026 10:30am