The FDA has approved the first two drugs for the rare genetic metabolic disorder Niemann–Pick disease, type C (NPC). Zevra Therapeutics’ arimoclomol (Miplyffa) is a once-rejected small ...
The recommended dosage is based on the patient’s body weight. The Food and Drug Administration (FDA) has approved Aqneursa ™ (levacetylleucine) for the treatment of neurological manifestations ...
The U.S. Food and Drug Administration has approved Zevra Therapeutics' Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination ...
IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has approved Aqneursa ...
Miplyffa is the first drug approved for treating associated neurological symptoms in adults and children 2 years and older. HealthDay News — The U.S. Food and Drug Administration has approved Miplyffa ...
“The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C," commented Laurie Turner, Family Services Manager at the National Niemann-Pick ...
Theranexus has developed the first oral solution of miglustat tailored to the needs of patients with Niemann-Pick type C disease, Theranexus is preparing to register this formulation with the EMA ...