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Research article

First published 2019

Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy

Michelle Pacione, Carly E. Siskind, John W. Day [...]

Journal of Neuromuscular Diseases

Retricted access

Review article

First published 2019-1

Nusinersen: A Treatment for Spinal Muscular Atrophy

Melanie K. Claborn, Debra L. Stevens, Cheri K. Walker [...]

Annals of Pharmacotherapy

Open access

Review article

First published 2020

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Ravindra N Singh, Eric W Ottesen, Natalia N Singh

Neuroscience Insights

Retricted access

Research article

First published 2022-6

Computed tomography-guided transforaminal lumbar puncture using local anesthesia and a straight 22-gauge spinal needle for intrathecal nusinersen in adults: Findings in 77 procedures

Mougnyan Cox, Kofi-Buaku Atsina, Preethi Ramchand [...]

Interventional Neuroradiology

Open access

Review article

First published 2026-3

Real-world evidence on nusinersen treatment of persons with SMA: a focused review

Susan E Matesanz, Richard S Finkel

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2018-04

Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease Patients

Willeke M.C. van Roon-Mom, Raymund A.C. Roos, Susanne T. de Bot

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2021-8

Strategies of stratification: Regulating market access in the era of personalized medicine

Sarah Wadmann, Amalie Martinus Hauge

Social Studies of Science

Retricted access

Review article

First published 2023-03

An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy

Yueyi Li, Hongyu Zeng, Yuhao Wei [...]

Human Gene Therapy

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