Within the past month, the FDA has announced the approval of new drugs to treat the rare disease – Niemann-Pick Type C. You ...

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 FDA recently authorized two drugs, MIPLYFFA (arimoclomol) and AQNEURSA (levacetylleucine) that are supposed to help stop ...

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 ...

October 19 is Niemann-Pick Day, focused on raising awareness about this rare genetic disease affecting children's metabolism ...

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 ...