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
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M. Patterson | B. Bembi | M. Gautschi | S. Day | S. Santra | A. Tylki-Szymańska | F. Deodato | A. Roubertie | P. Harmatz | C. Dali | E. Maier | E. Mengel | S. Grunewald | Nikolaj H. T. Petersen | S. Grønborg | M. Toro | L. Ingemann | Thomas Kirkegaard Jensen | B. Héron-Longe