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rare diseases
Pharma
FDA expands AZ's Ultomiris to NMOSD after prior rejection
AstraZeneca had to fight off a prior FDA complete response letter to get Ultomiris its NMOSD nod.
Angus Liu
Mar 25, 2024 10:31am
Enspryng falls short of Roche's expectations in myasthenia gravis
Mar 21, 2024 11:28am
Kyowa's Orchard hopes $4.25M gene therapy price tag bears fruit
Mar 20, 2024 10:40am
Orchard nabs approval for first MLD gene therapy in US
Mar 18, 2024 3:25pm
Charles River to help Navega produce gene therapy candidate
Mar 14, 2024 9:50am
Alnylam embarks on campaign to drive hATTR amyloidosis diagnoses
Mar 13, 2024 10:30am