Treatment Options for Niemann-Pick Type C
Por um escritor misterioso
Descrição
Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, describes dif
Gene therapy shows promise for treating Niemann-Pick disease type C1
Imaging of neuroinflammation in adult Niemann-Pick type C disease
Niemann-Pick type C: contemporary diagnosis and treatment of a classical disorder
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Niemann- Pick Disease Type C (NPC) Market Report 2023-2033 - Wissen Research
Treatment Options for Niemann-Pick Type C
Summer 2022 Newsletter – NNPDF
The Neuropsychiatry of Niemann-Pick Type C Disease in Adulthood
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Patient with Niemann-Pick disease type C: over 20 years' follow-up
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited
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