Acknowledgements
Niemann-Pick Disease, Fundación Niemann-Pick de España (Spain),, Associazione Italiana Niemann Pick, Niemann-Pick disease
NIEMANN-PICK DISEASE, NIEMANN-PICK, NIEMANN-PICK DISEASE
We thank each of the study sites and principal investigators participating in the IB1001-301 clinical trial for their participation. We thank the multinational Patient Organizations representing the NPC community for their essential involvement, including the following NPC Organizations: Australian Niemann-Pick Type C Foundation, Niemann-Pick Canada, Fundación Niemann-Pick de España (Spain), Associazione Italiana Niemann Pick (Italy), Volwassenen, Kinderen en Stofwisselingsziekten (VKS), International Niemann-Pick disease Alliance (Worldwide), National Niemann-Pick Disease Foundation (USA), Niemann-Pick Association of Fuenlabrada (Spain), Niemann-Pick Selbsthilfegruppe (Germany), Niemann-Pick Suisse (Switzerland), Niemann-Pick UK (UK), the Firefly Foundation (USA), and the Ara Parseghian Medical Research Foundation (USA). Finally, we are very grateful to all the patients and their families who participated in these studies.