Association Française Niemann Pick
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Association Française Niemann Pick
RareMD - Rare Disease Organizations
NPRF – Niemann-Pick Research Foundation
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
Different Niemann-Pick C1 Genotypes Generate Protein Phenotypes that Vary in their Intracellular Processing, Trafficking and Localization
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates
Social and medical needs of rare metabolic patients: results from a MetabERN survey, Orphanet Journal of Rare Diseases
Defective Schwann cell lipid metabolism alters plasma membrane dynamics in Charcot-Marie-Tooth disease 1A
Association Française Niemann Pick
Wnt5a Promotes Lysosomal Cholesterol Egress and Protects Against Atherosclerosis
Advocacy – NNPDF
Association française Niemann Pick
Defective Schwann cell lipid metabolism alters plasma membrane dynamics in Charcot-Marie-Tooth disease 1A
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por adulto (o preço varia de acordo com o tamanho do grupo)