Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy | Zendy

Eugenio Mercuri | Zendy; Basil T. Darras | Zendy; Claudia A. Chiriboga | Zendy; John Day | Zendy; Craig Campbell | Zendy; Anne M. Connolly | Zendy; Susan T. Iannaccone | Zendy; Janbernd Kirschner | Zendy; Nancy L. Kuntz | Zendy; Kayoko Saito | Zendy; Perry B. Shieh | Zendy; M. Tulinius | Zendy; Elena Mazzone | Zendy; Jacqueline Montes | Zendy; Kathie M. Bishop | Zendy; Qingqing Yang | Zendy; Richard Foster | Zendy; Sarah Gheuens | Zendy; C. Frank Bennett | Zendy; Wildon Farwell | Zendy; Eugene Schneider | Zendy; Darryl C. De Vivo | Zendy; Richard S. Finkel | Zendy

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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Author(s) -

Eugenio Mercuri,

Basil T. Darras,

Claudia A. Chiriboga,

John Day,

Craig Campbell,

Anne M. Connolly,

Susan T. Iannaccone,

Janbernd Kirschner,

Nancy L. Kuntz,

Kayoko Saito,

Perry B. Shieh,

M. Tulinius,

Elena Mazzone,

Jacqueline Montes,

Kathie M. Bishop,

Qingqing Yang,

Richard Foster,

Sarah Gheuens,

C. Frank Bennett,

Wildon Farwell,

Eugene Schneider,

Darryl C. De Vivo,

Richard S. Finkel

Publication year - 2018

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa1710504

Subject(s) - spinal muscular atrophy , medicine , physical medicine and rehabilitation , atrophy , progressive muscular atrophy , physical therapy , disease , amyotrophic lateral sclerosis

Nusinersen is an antisense oligonucleotide drug that modulates pre-messenger RNA splicing of the survival motor neuron 2 ( SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA).

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