A Gene Transfer Study for Late-Onset Pompe Disease

2019-09-23 04:51:41 | BioPortfolio


The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

Study Design


Pompe Disease




Not yet recruiting


Spark Therapeutics

Results (where available)

View Results


Published on BioPortfolio: 2019-09-23T04:51:41-0400

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Medical and Biotech [MESH] Definitions

Animate or inanimate sources which normally harbor disease-causing organisms and thus serve as potential sources of disease outbreaks. Reservoirs are distinguished from vectors (DISEASE VECTORS) and carriers, which are agents of disease transmission rather than continuing sources of potential disease outbreaks.

Analyses for a specific enzyme activity, or of the level of a specific enzyme that is used to assess health and disease risk, for early detection of disease or disease prediction, diagnosis, and change in disease status.

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