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A Multicenter, Multinational, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human Alpha-L-Iduronidase in Patients with Mucopolysaccharidosis I. (ALID 006-01)

The objective of this extension study is to collect additional long-term efficacy and safety data of rh α-L-iduronidase in patients with MPS-I disease who previously were treated under a double-blind protocol (Study Number ALID-003-99). This is a multi-center, open-label extension study. Closed to enrollment.

Multicenter, Open-label Study of the Safety and Efficacy of Fabrazyme® in patients with Fabry Disease that previously participated in the AGAL-008-00 Study (AGAL02503) Final: 14 August 2003

This study is designed to further determine the safety and effectiveness of a medicine called Fabrazyme® (also referred to as "recombinant human α-galactosidase A" or "r-hαGAL") to treat Fabry disease. This is a multi-center, open-label extension study. Closed to enrollment.

A Randomized, Open-Label, Multicenter, Multinational, Dose-Ranging Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA)

Treatment in Patients less than or equal to 6 Months Old with Infantile-Onset Pompe Disease (Glycogen Storage Disease Type II) Protocol # AGLU01602. The study is designed to evaluate the long-term safety and potential benefit of an investigational medication called recombinant human acid alpha Glucosdidase (rhGAA) in patients with Pompe Disease. This is a multi-center, open-label study. Closed to enrollment.

Expanded access use of Recombinant Human Acid Alpha-Glucosidase (rhGAA)(Myozyme) in Patients with Infantile-Onset Pompe Disease. (AGLU02203)

This expanded access program was designed by the sponsor to provide recombinant Human Acid alpha Glucosidase (rhGAA, MyozymeT), which is a purified human enzyme produced in Chinese Hamster Ovary (CHO) cells, to patients who are severely affected with infantile onset Pompe disease and are ineligible to take part in any other treatment studies for Pompe disease. This program is still enrolling.

A Phase IV, Multicenter, Randomized, Dose Frequency Study of the Safety and Efficacy of Cerezyme® Infusions Every Four Weeks Versus Every Two Weeks in the Maintenance Therapy of Patients with Type 1 Gaucher Disease

Most patients with Gaucher disease that are symptomatic receive Cerezyme® infusions once every two weeks. This treatment schedule is considered standard of care. The main purpose of this study is to determine whether the health status of patients with Type 1 Gaucher disease can be maintained if they are given their total 4-week dose of Cerezyme® in one infusion every four weeks (instead of their total 4-week dose split into two infusions, one infusion every two weeks). The study will monitor patient's health and how they feel on the less frequent infusion schedule and whether this new schedule provides greater convenience for patients and less disruption to their work schedules and personal lives. This is a multicenter study. Enrollment at Emory to begin August 2004.

Fabry Registry Protocol

The Fabry Registry is an ongoing, post marketing observational database that tracks the natural history and outcomes of patients with Fabry disease. Participation is open to all patients with Fabry disease. Physicians are encouraged to collaborate, share observations, and generate hypotheses for evaluation, as well as assist in the collection of clinical data in an effort to guide and assess future therapeutic interventions. This is a world-wide, multi-center Registry.

International Collaborative Gaucher Group (ICGG) Gaucher Registry Protocol

The Gaucher Registry is an ongoing, post-marketing, observational database that tracks outcomes of routine clinical practice for patients with Gaucher disease. It is open to all Gaucher patients. All physicians participating in the Registry are considered members of the International Collaborative Gaucher Group (ICGG). Data collected from ICGG physicians will represent Gaucher disease practice patterns under common clinical conditions. Thus, the data collected by this international, collaborative Registry will provide information to better characterize the natural history and progression of Gaucher disease, as well as the clinical responses of patients whose physicians have prescribed ERT. This is a world wide, multi-center Registry.

MPS 1 Registry Protocol

The MPS I Registry is an ongoing, post marketing observational database that tracks the natural history and outcomes of patients with MPS I disease. Participation is open to all g patients with MPS I disease. Physicians are encouraged to collaborate, share observations, and generate hypotheses for evaluation, as well as assist in the collection of clinical data in an effort to guide and assess future therapeutic interventions. This is a world wide, multi-center Registry.