Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C

Author:

Bremova-Ertl Tatiana1ORCID,Ramaswami Uma1ORCID,Brands Marion1ORCID,Foltan Tomas1,Gautschi Matthias1ORCID,Gissen Paul1ORCID,Gowing Francesca1,Hahn Andreas1ORCID,Jones Simon1,Kay Richard1,Kolnikova Miriam1ORCID,Arash-Kaps Laila1,Marquardt Thorsten1ORCID,Mengel Eugen1ORCID,Park Julien H.1ORCID,Reichmannová Stella1ORCID,Schneider Susanne A.1ORCID,Sivananthan Siyamini1ORCID,Walterfang Mark1ORCID,Wibawa Pierre1ORCID,Strupp Michael1ORCID,Martakis Kyriakos1ORCID

Affiliation:

1. From University Hospital Bern, Bern, Switzerland (T.B.-E., M.G.); Royal Free London NHS Foundation Trust (U.R., F.G.), University College London (U.R.), and Great Ormond Street Hospital, University College London (P.G., S.S.), London, Royal Manchester Children’s Hospital, University of Manchester, Manchester (S.J.), and RK Statistics, Bakewell (R.K.) — all in the United Kingdom; Emma Children’s Hospital–Amsterdam, University Medical Center, Amsterdam (M.B.); the National Institute of Children’s Diseases,...

Funder

IntraBio Inc.

Publisher

Massachusetts Medical Society

Subject

General Medicine

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