Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Authors

Eugenio Mercuri

Published

February 14, 2018

Abstract

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).

Supporters

Support the authors with ResearchCoin

Journal

New England Journal of Medicine

Topics

DOI

10.1056/nejmoa1710504

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Authors

Eugenio Mercuri

Published

February 14, 2018

Abstract

Supporters

Support the authors with ResearchCoin

Journal

New England Journal of Medicine

Topics

DOI

10.1056/nejmoa1710504

Paper

Paper

Paper

Paper

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Abstract

Supporters

Journal

Topics

DOI

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Abstract

Supporters

Journal

Topics

DOI