Deficiency
BioMarin Acquires Inozyme Pharma for $270M, Gaining Phase 3 Rare Disease Therapy
BioMarin; Inozyme Pharma; acquisition; rare disease; INZ-701; ENPP1 Deficiency; enzyme replacement therapy; Phase 3 clinical trial
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder
Agios’ Pyrukynd Misses Primary Endpoint in First Pediatric Study for Rare Blood Disorder
Agios Pharmaceuticals, Pyrukynd, mitapivat, PK deficiency, pediatric study, blood disorder, Phase 3 ACTIVATE-KidsT study