Sanfilippo syndrome registry project and natural history studies: an example of patients, parents and researchers collaborating for a cure

Abstract

In the past 5 years two clinical trials for Sanfilippo type A have been conducted, namely Shire’s enzyme replacement therapy and Lysogene’s gene therapy. Nationwide Children’s Hospital is aiming to start gene-therapy clinical trials for types A & B in 2014. For other Sanfilippo types C and D the research is at preclinical stages including genetherapy (Type… (More)

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