Patient-derived Nerve Cells May Help Advance Pompe Research

Patient-derived Nerve Cells May Help Advance Pompe Research

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Patient-derived Nerve Cells May Help Advance Pompe Research

Newly developed nerve cells derived from patients could be used as a model to study neurological involvement in Pompe disease and test potential therapies, a study reports. Notably, data from these models suggested that a combination of two molecules, which was previously shown to have beneficial effects in models of other lysosomal storage disorders, should be further evaluated as a potential treatment for Pompe disease. The study, “Modeling CNS Involvement in Pompe Disease Using Neural…

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