Therapeutic efficacy of 3,4-Diaminopyridine phosphate on neuromuscular junction in Pompe disease

Therapeutic efficacy of 3,4-Diaminopyridine phosphate on neuromuscular junction in Pompe disease

3,4-Diaminopyridine (3,4-DAP) and its phosphate form, 3,4-DAPP have been used efficiently in the past years to treat muscular weakness in myasthenic syndromes with neuromuscular junctions (NMJs) impairment. Pompe disease (PD), an autosomal recessive metabolic disorder due to a defect of the lysosoma...

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Bibliographic Details
Main Authors: Cinzia Bragato, Flavia Blasevich, Gary Ingenito, Renato Mantegazza, Lorenzo Maggi
Format: Article
Language:English
Published: Elsevier 2021-05-01
Series:Biomedicine & Pharmacotherapy
Subjects:
Pompe disease
Acid α-glucosidase
3,4-Diaminopyridine phosphate (3,4-DAPP)
Zebrafish
Neuromuscular junction (NMJ)
Online Access:http://www.sciencedirect.com/science/article/pii/S0753332221001426

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