This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The objectives of this study are to continue treatment of three patients with Fabry disease with enzyme replacement therapy (ERT) using Replagal (agalsidase alfa) at a dose of 0.2mg/kg of body weight administered every 2 weeks. These patients were previously on clinical research protocol 04-N-0027 at the National Institute of Neurological Disorders and Stroke (NINDS) under P.I. Dr. Raphael Schiffmann. With the closure of protocol 04-N-0027 the patients are being transitioned to this study. Dr. Tifft will assume Dr. Schiffmann's IND for Replagal. This open-label study will monitor safety and outcome measures of disease stability as part of the Fabry Outcome Survey (FOS), an international registry of outcome data on patients treated with Replagal. 04-N-0027 was an investigator-sponsored protocol funded by Transkaryotic Therapies (TKT), now Shire, Human Genetic Therapies (HGT), and all TKT protocols and obligations were assumed by Shire HGT. The hypothesis is that patients currently stable on over 5 years of Replagal infusiona at 0.2mg/kg body weight every other week will remain stable. Safety profile of the drug will continue to be favorable.
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