Yahoo Finance

Niemann-Pick Disease Type C Market is Expected to Showcase Significant Growth at a CAGR of 14.8% During the Forecast Period (2025–2034) | DelveInsight

DelveInsight’s Niemann-Pick Disease Type C Market Insights report includes a comprehensive understanding of current treatment practices, emerging Niemann-Pick disease type C drugs, market share of ...
Business Wire

Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease ...

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...
Healio

Miplyffa commercially available for treatment of Niemann-Pick disease type C

Please provide your email address to receive an email when new articles are posted on . Zevra Therapeutics has announced that Miplyffa, the first Nieman-Pick disease type C treatment approved by the ...
Healio

FDA approves Miplyffa for Niemann-Pick disease type C

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 ...
Nasdaq

Zevra Launches Niemann-Pick Disease Type C Drug Miplyffa In The U.S.

(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 ...
Morningstar

Mandos Health to Present New Analyses in Niemann-Pick Disease Type C at the 2025 American Neurological Association Annual Meeting

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 ...
Morningstar

AQNEURSA® (levacetylleucine) Recommended for EU Approval by the CHMP to Treat Niemann-Pick Disease Type C

IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products ...
PharmiWeb

Data Analyses Show Benefit for Individuals With Infantile-Onset Niemann-Pick Disease Type C Treated With Investigational Drug Adrabetadex

Analysis shows statistically significant survival benefit in individuals with infantile-onset Niemann-Pick disease type C (NPC) treated with adrabetadex compared to matched external controls Findings ...
Business Wire

Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros B.V. today announced positive topline data from its RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat in patients with a genetic ...
Mena FN

Niemann-Pick Disease Type C Market Is Expected To Showcase Significant Growth At A CAGR Of 14.8% During The Forecast Period (20252034) Delveinsight

Niemann Pick Disease Type A Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the ...
Monthly Prescribing Reference

Miplyffa Approved for Niemann-Pick Disease Type C

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) ...