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

Bragato, Cinzia; Blasevich, Flavia; Ingenito, Gary; Mantegazza, Renato; Maggi, Lorenzo

Bragato, Cinzia; Blasevich, Flavia; Ingenito, Gary; Mantegazza, Renato; Maggi, Lorenzo.

Afiliación

Bragato C; Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, Milan 20133, Italy. Electronic address: cinzia.bragato@istituto-besta.it.
Blasevich F; Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, Milan 20133, Italy.
Ingenito G; Catalyst Pharmaceuticals Inc, Coral Gables, FL, USA.
Mantegazza R; Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, Milan 20133, Italy.
Maggi L; Neuromuscular Diseases and Neuroimmunology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, Milan 20133, Italy.

Biomed Pharmacother ; 137: 111357, 2021 05.

Article en En | MEDLINE | ID: mdl-33724918

Asunto(s)

Amifampridina/uso terapéutico; Enfermedad del Almacenamiento de Glucógeno Tipo II/tratamiento farmacológico; Unión Neuromuscular/efectos de los fármacos; Animales; Conducta Animal; Embrión no Mamífero; Actividad Motora/efectos de los fármacos; Fibras Musculares Esqueléticas/efectos de los fármacos; Unión Neuromuscular/ultraestructura; Receptores Colinérgicos/efectos de los fármacos; Pez Cebra; alfa-Glucosidasas/metabolismo

Palabras clave

3,4-Diaminopyridine phosphate (3,4-DAPP); Acid α-glucosidase; Neuromuscular junction (NMJ); Pompe disease; Zebrafish

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedad del Almacenamiento de Glucógeno Tipo II / Amifampridina / Unión Neuromuscular Idioma: En Revista: Biomed Pharmacother Año: 2021 Tipo del documento: Article

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