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

Compartilhe seus vídeos com amigos, familiares e todo o mundo

Fig 2., Subcortical Volumetric Reductions in Adult Niemann-Pick Disease Type C: A Cross-Sectional Study

Niemann-Pick disease: Causes, symptoms, treatment, and more

Niemann-Pick Type C Disease - Pipeline Insight, 2021

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

Rare Disease Video - Niemann-Pick Disease Types A & B - National Organization for Rare Disorders

FDA needs to be more flexible assessing rare disease treatments - STAT

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other

Drug Repurposing for Rare Diseases: Trends in Pharmacological Sciences

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

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

Niemann-Pick disease type C clinical database: cognitive and coordination deficits are early disease indicators – topic of research paper in Clinical medicine. Download scholarly article PDF and read for free on CyberLeninka

Combination therapy a potential strategy for treating Niemann-Pick disease

Case Studies, Clinical Development Case Studies

Advancements in Niemann-Pick Disease Type C Treatments