Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience.

One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better? / Ruggiero, Lucia; Iodice, Rosa; Esposito, Marcello; Dubbioso, Raffaele; Tozza, Stefano; Vitale, Floriana; Santoro, Lucio; Manganelli, Fiore. - In: THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS. - ISSN 1756-2856. - 11:(2018), p. 1756286418809588. [10.1177/1756286418809588]

One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better?

Ruggiero, Lucia;Iodice, Rosa;Esposito, Marcello;Dubbioso, Raffaele;Tozza, Stefano;Vitale, Floriana;Santoro, Lucio;Manganelli, Fiore
2018

Abstract

Ataluren was approved for the treatment of nmDMD, both the efficacy and safety have been previously reported only from clinical trials but no report exists about real-life experience.
2018
One-year follow up of three Italian patients with Duchenne muscular dystrophy treated with ataluren: is earlier better? / Ruggiero, Lucia; Iodice, Rosa; Esposito, Marcello; Dubbioso, Raffaele; Tozza, Stefano; Vitale, Floriana; Santoro, Lucio; Manganelli, Fiore. - In: THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS. - ISSN 1756-2856. - 11:(2018), p. 1756286418809588. [10.1177/1756286418809588]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/728106
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