Efficacy and Safety of N-Acetyl-L-Leucine in Children and Adults With GM2 Gangliosidoses.

Kyriakos MartakisJens ClaassenJordi Gascon-BayariNicolina GoldschaggAndreas HahnAnhar HassanAnita HennigSimon JonesRichard KayHeather LauSusan PerlmanReena SharmaSusanne A Schneider Tatiana Bremova-Ertl

Published in: Neurology (2022)

The trial is registered with ClinicalTrials.gov (NCT03759665; registered 30-Nov-2018), EudraCT (2018-004406-25), and DRKS (DRKS00017539). The first patient was enrolled 07-June-2019.

Keyphrases

young adults
case report
study protocol
phase iii
clinical trial
phase ii
randomized controlled trial
open label