Latest News for: niemann-pick disease

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Niemann-Pick Disease Type C NPC Market Analysis and Forecast, 2025-2035 | Personalized Medicine and Gene ...

Business Wire 13 Aug 2025
DUBLIN--(BUSINESS WIRE)--The "Niemann-Pick Disease Type C NPC Market - A Global and Regional ...
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Niemann-Pick Disease Type C NPC Market Research 2025-2035 | Emerging Gene Therapies Offer New Hope ...

Nasdaq Globe Newswire 01 Aug 2025
Niemann-Pick Disease Type C (NPC) is a rare genetic disorder caused by mutations in the NPC1 or NPC2 genes, leading to harmful lipid accumulation in organs like the liver and brain ...
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AQNEURSA® (levacetylleucine) Recommended for EU Approval by the CHMP to Treat Niemann-Pick Disease Type C

The Galveston Daily News 31 Jul 2025
AUSTIN, Texas--(BUSINESS WIRE)--Jul 30, 2025-- ....
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Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type ...

The Galveston Daily News 28 Jul 2025
LEIDEN, Netherlands--(BUSINESS WIRE)--Jul 28, 2025-- ....
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Niemann-Pick Disease Market Forecast 2034: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Statistics, Revenue, Treatment, ...

GetNews 16 Jul 2025
(Albany, USA) DelveInsight has recently released epidemiology-based market reports focusing on Niemann-Pick disease, including Niemann-Pick Disease A/B and Niemann-Pick Disease C ... Disease Type C (NPC).
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Zevra Therapeutics Announces MIPLYFFA� (arimoclomol) Featured in Presentations at the National Niemann Pick Disease Foundation Conference

Nasdaq Globe Newswire 11 Jul 2025
Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C (NPC).
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Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease ...

Nasdaq Globe Newswire 18 Jun 2025
Phase 3 TransportNPC study to continue based on the independent DMC review of safety and efficacy data at prespecified 48-week interim analysis ... .
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Niemann-Pick Disease Market Insights: Key Trends and Developments Across Different Types of Niemann-Pick Disease | DelveInsight

Nasdaq Globe Newswire 02 Jun 2025
According to DelveInsight’s analysis, the market for Niemann-Pick disease is anticipated to ...
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Niemann-Pick Disease Type C Market to Expand Significantly by 2034, States DelveInsight Report | Zevra ...

GetNews 23 May 2025
DelveInsight’s “Niemann-Pick Disease Type C Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Niemann-Pick Disease Type C, ...
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