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Related Experiment Video

Updated: Feb 7, 2026

Accessing the Subdural Space of the Rodent Spinal Cord for Treatment Delivery

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Nusinersen: A Treatment for Spinal Muscular Atrophy.

Melanie K Claborn¹, Debra L Stevens¹, Cheri K Walker¹

¹1 Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.

The Annals of Pharmacotherapy

|July 17, 2018

Summary

Nusinersen (Spinraza) is a safe and effective treatment for spinal muscular atrophy (SMA), improving motor function in patients. While well-tolerated, its administration and cost present clinical challenges.

Keywords:

Spinraza nusinersen spinal muscular atrophy

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Area of Science:

Neurology
Genetics
Pharmacology

Background:

Spinal muscular atrophy (SMA) is a genetic disorder characterized by motor neuron loss.
SMA is caused by mutations in the 5q chromosome, leading to survival motor neuron protein deficiency.
Nusinersen is the first approved drug therapy for SMA.

Purpose of the Study:

To review the efficacy and safety of nusinersen (Spinraza) in treating spinal muscular atrophy (SMA).

Main Methods:

A comprehensive literature search of PubMed and MEDLINE was conducted.
Included data from clinical trials, manufacturer information, and clinicaltrials.gov.
All identified clinical trials of nusinersen were analyzed.

Main Results:

Nusinersen demonstrated improvements in motor function across all types of SMA.
Common adverse effects included respiratory infections, headache, and back pain.
Phase III trial data indicated positive changes in the clinical course of SMA patients.

Conclusions:

Nusinersen is a safe and effective treatment for SMA, well-tolerated in all age groups.
Challenges in clinical practice include intrathecal delivery and high cost.
The drug offers a significant advancement in SMA patient care.