Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial | Zendy

Laurent Servais | Zendy; Eugenio Mercuri | Zendy; Volker Straub | Zendy; Michela Guglieri | Zendy; Andreea Seferian | Zendy; Mariacristina Scoto | Zendy; Daniela Leone | Zendy; E. Koenig | Zendy; Navid Khan | Zendy; A. Dugar | Zendy; Xiaodong Wang | Zendy; Baoguang Han | Zendy; Dan Wang | Zendy; Francesco Muntoni | Zendy; Chiara Brusa | Zendy; Laura Antonaci | Zendy; Claudia Brogna | Zendy; Laura Merli | Zendy; Mauro Monforte | Zendy; Giulia Norcia | Zendy; Marika Pane | Zendy; Gloria Ferrantini | Zendy; George Dickson | Zendy; Jennifer E. Morgan | Zendy; Valentina Sardone | Zendy; Cody Akana | Zendy; Jay S. Charleston | Zendy; Cody A. Desjardins | Zendy; Saleh El-Husayni | Zendy; Diane E. Frank | Zendy; Frederick J. Schnell | Zendy

Open Access

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Author(s) -

Laurent Servais,

Eugenio Mercuri,

Volker Straub,

Michela Guglieri,

Andreea Seferian,

Mariacristina Scoto,

Daniela Leone,

E. Koenig,

Navid Khan,

A. Dugar,

Xiaodong Wang,

Baoguang Han,

Dan Wang,

Francesco Muntoni,

Chiara Brusa,

Laura Antonaci,

Claudia Brogna,

Laura Merli,

Mauro Monforte,

Giulia Norcia,

Marika Pane,

Gloria Ferrantini,

George Dickson,

Jennifer E. Morgan,

Valentina Sardone,

Cody Akana,

Jay S. Charleston,

Cody A. Desjardins,

Saleh El-Husayni,

Diane E. Frank,

Frederick J. Schnell

Publication year - 2021

Publication title -

nucleic acid therapeutics

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 2.255

H-Index - 67

eISSN - 2159-3345

pISSN - 2159-3337

DOI - 10.1089/nat.2021.0043

Subject(s) - duchenne muscular dystrophy , medicine , ambulatory , term (time) , exon skipping , muscular dystrophy , open label , exon , physical medicine and rehabilitation , bioinformatics , adverse effect , pharmacology , genetics , biology , gene , alternative splicing , physics , quantum mechanics

The content you want is available to Zendy users.

Already have an account? Click here to sign in.

Having issues? You can contact us here

Accelerating Research