LEMS Therapy Eases Nerve-Muscle Problems in Pompe Zebrafish Model

LEMS Therapy Eases Nerve-Muscle Problems in Pompe Zebrafish Model

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LEMS Therapy Eases Nerve-Muscle Problems in Pompe Zebrafish Model

Administration of 3,4-diaminopyridine phosphate (3,4-DAPP) eased problems at the neuromuscular junction (NMJ) — sites of nerve-muscle communication — in a zebrafish model of Pompe disease. Results suggest that the medication, also known as amifampridine and approved under the brand names Firdapse and Ruzurgi for Lambert-Eaton myasthenic syndrome, a NMJ disorder, may have potential in treating Pompe patients.  “We can hypothesize that the 3,4-DAPP treatment not only has a beneficial action on already formed NMJs, but can have a…

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