Browsing Tag
Fragile X syndrome
3 posts
Mirum Pharmaceuticals (NASDAQ: MIRM) jumps 13% after VISTAS Phase 2b PSC win
Two Phase 2b wins in seven days. A pre-NDA meeting this summer. Mirum is starting to look less like a single-product rare disease story.
ZYN002 misses pivotal Fragile X trial — What does the setback mean for Harmony’s pipeline and stock outlook?
Harmony’s ZYN002 fails key Fragile X trial, sending HRMY stock lower. Find out why placebo effects mattered and what’s next for its pipeline.
Quiver Bioscience joins forces with Dup15q Alliance to develop ASO therapy for rare neurological disorder
Quiver Bioscience partners with the Dup15q Alliance to advance antisense oligonucleotide therapy for Dup15q syndrome. Discover how AI-driven research is shaping genetic medicine.