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1d
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive ...
FiercePharma
3d
AstraZeneca's Fasenra plays catch-up with GSK's Nucala, gaining FDA nod for rare autoimmune disease
Another key difference in the study was the dosing. While patients on Fasenra received one 30-mg subcutaneous injection every ...
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