December 27, 2023 at 7:30 AM EST CELEBRATION, Fla., Dec. 27, 2023 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based …
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NPC
Azafaros’ Phase 2 RAINBOW study for nizubaglustat in NPC and GM2 patients fully enrolled
December 11, 2023 Leiden, The Netherlands, 11 December 2023 – Azafaros B.V. today announces that it has exceeded the target number of patients in the enrollment of its Phase 2 clinical trial, RAINBOW (NCT05758922), treating patients with severe rare metabolic diseases. The success in enrollment is a major milestone for Azafaros’ lead asset, nizubaglustat, which is …
IV Adrabetadex (cyclodextrin) Trial for Neonatal Liver Disease Started
Researchers from St. Louis Children’s Hospital at Washington University School of Medicine are enrolling infants 0-6 months with diagnosed NPC disease and evidence of liver disease. The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with NPC disease. The study has an initial 6-week phase during …