Efficacy and safety of arimoclomol in patients with...

Molecular Genetics and Metabolism 2020 / 02 Vol. 129; Iss. 2

Efficacy and safety of arimoclomol in patients with Niemann-Pick disease type C: Results from a double-blind, randomized placebo-controlled trial with a novel treatment

Patterson, Marc, Mengel, Eugen, Bembi, Bruno, Del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Harmatz, Paul, Heron-Longe, Benedicte, Maier, Esther M.,
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Volume:
129
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2019.11.331
Date:
February, 2020
File:
PDF, 323 KB
2020
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