The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease

Author:

Hammond Natalie,Munkacsi Andrew B.,Sturley Stephen L.

Funder

Ara Parseghian Medical Research Foundation

Dana's Angels Research Trust

Neurological Foundation of New Zealand

New Zealand Organisation of Rare Diseases

Actelion Pharmaceuticals

National Niemann-Pick Disease Foundation

National Institutes of Health

Publisher

Elsevier BV

Subject

Cell Biology,Molecular Biology

Reference191 articles.

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4. Consensus clinical management guidelines for Niemann-Pick disease type C;Geberhiwot;Orphanet J. Rare Dis.,2018

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