GAINESVILLE -
'We truly value the opportunity and are honored to present our data at this preeminent scientific congress to further strengthen our voice within the NPC community. Our TransportNPC study is the most comprehensive ongoing controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes for the treatment of Niemann-Pick Disease Type C1. We are extremely proud of the progress with our fully enrolled, ongoing TransportNPC. Our team has strong belief in our program, and we look forward to further engaging with the community at this important meeting and discussing our potential to have a meaningful impact for people living with NPC globally,' commented
Details of the poster presentations are as follows
Title: TRANSPORTNPC: Phase 3 global trial of intravenous hydroxypropyl-beta-cyclodextrin (HPCD) in patients three years of age or older with Niemann-Pick disease type C1 (NPC1)
Presenter:
About SSIEM
The aim of the Society is to foster the study of inherited metabolic disorders and related topics. The Society exists to promote exchange of ideas between professional workers in different disciplines who are interested in inborn errors of metabolism (IEM). This is achieved through the organization of the annual symposium and associated scientific meetings, publications and provision of funding for educational and training support.
The Society, a registered charity and limited company, accepts donations from sponsors sympathetic to its aims. The Council, within its financial resources, is willing to spread interest and study in inborn errors around the world by selectively supporting membership and attendance at conferences from areas of the world where financial resources are more limited. The Society does not directly fund research, but offers on request, advice to other organizations that do so. Education and training in IEM are also an important aim of the SSIEM.
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