Tag: Rare Disease
PharmaSignal — Rare Disease therapeutic area
Maze tumbles despite positive data for kidney disease drug
March 25, 2026
BioPharma Dive
Investors had reservations about data for a drug that could compete with a candidate from Vertex in APOL1-mediated kidney disease.
Beam posts positive data on base editing treatment for AATD
March 25, 2026
BioPharma Dive
Updated data showed the biotech’s approach to the rare lung and liver condition helped restore functional protective proteins in a small study.
Sarepta sees early success with RNAi drugs from Arrowhead
March 25, 2026
BioPharma Dive
Long-awaited results from Phase 1/2 studies lifted Sarepta s stock and, according to the company, suggest potential for two medicines to treat muscle-weakening conditions.