Could Actio’s KCNT1 epilepsy drug become an ultra-rare disease test case for the FDA?
Ultra-rare epilepsy has few paths forward. Actio’s ABS-1230 now tests whether precision genetics can reshape FDA evidence standards.
Travere Therapeutics’ $475 million raise puts spotlight on rare disease execution risk
Rare disease pipelines need time and cash. Travere’s $475 million debt deal shows how biotech balance sheets are being rebuilt.
Can Sarepta Therapeutics prove RNA interference works in FSHD1 and DM1 with early Phase 1/2 data
Read why Sarepta Therapeutics’ first siRNA data in FSHD1 and DM1 could matter for rare disease drug development and investor sentiment.
Can Pretzel Therapeutics’ PX578 finally address the root cause of POLG disease in mitochondrial DNA depletion syndromes?
Pretzel Therapeutics advances PX578 for POLG disease. Discover how mitochondrial DNA restoration could reshape treatment for rare mitochondrial disorders.
Phase 3 CYPRESS miss forces Theravance Biopharma to rethink pipeline and accelerate strategic alternatives
Theravance Biopharma halted its ampreloxetine program after a Phase 3 failure. Discover what the CYPRESS results mean for the company and the neurogenic hypotension market.
Why Beren Therapeutics’ adrabetadex NDA could redefine approval standards in ultra-rare pediatric disease
FDA acceptance of adrabetadex puts disease modification for infantile-onset NPC under review. See what this decision could change for rare disease approvals.