International Pompe Association

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"Genzyme, a Sanofi company, has a long history of involvement with Pompe disease and partnership with the community. This led to the availability of the first approved treatment, enzyme replacement therapy (ERT) in 2006. We recognize that unmet medical need doesn’t always mean developing a therapy for a disease with no treatment options, but can also mean improving upon an already existing treatment in a significant way...
Genzyme is in the early stages of investigating a next generation enzyme replacement therapy (neoGAA) for Pompe disease...
Currently there is an ongoing Phase I study known as NEO1, to evaluate the safety and efficacy of neoGAA dosing in adults with late‐onset Pompe disease...
The study is planned to enroll 21 adult patients with late‐onset Pompe disease, and will include patients who have never been treated with ERT and patients who have been previously treated with ERT for at least 9 months. NEO1 is designed to evaluate the safety and tolerability of 3 different doses of neoGAA...
The study is being conducted at 18 sites in the United States and Europe (UK, Netherlands, Belgium, Denmark, France, and Germany). For more information please ask your treating physician or visit www.clinicaltrials.gov (ClinicalTrials.gov identifier: NCT01898364) or in Europe visit www.clinicaltrialsregister.eu (EudraCT number 2012‐004167‐42)."

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