Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...
Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...
(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with ...
IntraBio’s Aqneursa receives European Commission’s marketing approval for treatment of Niemann-Pick Type C disease: Austin, Texas Saturday, January 24, 2026, 14:00 Hrs [IST] I ...
CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick ...
Mandos Health® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that new analyses detailing the clinical and biological effects of its investigational drug, adrabetadex, in ...
The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the genetic disorder Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative ...
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 of ...
Please provide your email address to receive an email when new articles are posted on . The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated ...
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