Rare Diseases Research: Clinical Trial for Niemann-Pick Type C
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Last updated 12 abril 2025

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Niemann-Pick Type C News Dana's Angels Research Trust (DART)

New hope for Niemann-Pick Disease Type C treatment as Bloomsbury Genetic Therapies receives Orphan Drug Designations from the European Commission and US FDA - 17 - 10 - 2023 - UCLB

Combination therapy a potential strategy for treating Niemann-Pick disease

Clinical patient groups with an increased risk of Niemann-Pick disease

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

PDF) Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

Gene therapy shows promise for treating Niemann-Pick disease type C1

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

Speaker Series - Niemann-Pick Disease Type C
Think Again. Think NP-C

At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

Newborn screening test developed for rare, deadly neurological disorder – Washington University School of Medicine in St. Louis
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