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Nusinersen: A Treatment for Spinal Muscular Atrophy.
Ann Pharmacother 2019; 53(1):61-69AP

Abstract

OBJECTIVE

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

DATA SOURCES

An English-language literature search of PubMed and MEDLINE (1946 to June 2018) was performed using the terms nusinersen, ISIS-SMN (Rx), and spinal muscular atrophy. Manufacturer prescribing information, abstracts, article bibliographies, and clinicaltrials.gov data were incorporated for additional materials.

STUDY SELECTION/DATA EXTRACTION

All clinical trials of nusinersen were identified and analyzed in the review.

DATA SYNTHESIS

Nusinersen is the first drug therapy approved for the treatment of SMA. It is a novel modified antisense oligonucleotide designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency. Nusinersen has been studied for safety, pharmacokinetics, and efficacy in both open-label and randomized controlled trials. The studies show improvement in motor function across SMA of all types. The most common adverse effects were respiratory tract infections, headache, back pain, constipation, and post-lumbar puncture syndrome. Relevance to Patient Care and Clinical Practice: Based on phase III trial data, nusinersen produced positive changes in the clinical course of patients with SMA. The acquisition and administration of nusinersen present a number of challenges in clinical practice. Its intrathecal delivery and costly price tag must be recognized.

CONCLUSION

Nusinersen is safe and effective in patients with SMA. It was well tolerated across all studied age groups.

Authors+Show Affiliations

1 Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.1 Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.1 Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.1 Southwestern Oklahoma State University College of Pharmacy, Weatherford, OK, USA.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

30008228

Citation

Claborn, Melanie K., et al. "Nusinersen: a Treatment for Spinal Muscular Atrophy." The Annals of Pharmacotherapy, vol. 53, no. 1, 2019, pp. 61-69.
Claborn MK, Stevens DL, Walker CK, et al. Nusinersen: A Treatment for Spinal Muscular Atrophy. Ann Pharmacother. 2019;53(1):61-69.
Claborn, M. K., Stevens, D. L., Walker, C. K., & Gildon, B. L. (2019). Nusinersen: A Treatment for Spinal Muscular Atrophy. The Annals of Pharmacotherapy, 53(1), pp. 61-69. doi:10.1177/1060028018789956.
Claborn MK, et al. Nusinersen: a Treatment for Spinal Muscular Atrophy. Ann Pharmacother. 2019;53(1):61-69. PubMed PMID: 30008228.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Nusinersen: A Treatment for Spinal Muscular Atrophy. AU - Claborn,Melanie K, AU - Stevens,Debra L, AU - Walker,Cheri K, AU - Gildon,Brooke L, Y1 - 2018/07/16/ PY - 2018/7/17/pubmed PY - 2018/7/17/medline PY - 2018/7/17/entrez KW - KW - Spinraza KW - nusinersen KW - spinal muscular atrophy SP - 61 EP - 69 JF - The Annals of pharmacotherapy JO - Ann Pharmacother VL - 53 IS - 1 N2 - OBJECTIVE: To review the efficacy and safety of nusinersen (Spinraza) in the treatment of spinal muscular atrophy (SMA). DATA SOURCES: An English-language literature search of PubMed and MEDLINE (1946 to June 2018) was performed using the terms nusinersen, ISIS-SMN (Rx), and spinal muscular atrophy. Manufacturer prescribing information, abstracts, article bibliographies, and clinicaltrials.gov data were incorporated for additional materials. STUDY SELECTION/DATA EXTRACTION: All clinical trials of nusinersen were identified and analyzed in the review. DATA SYNTHESIS: Nusinersen is the first drug therapy approved for the treatment of SMA. It is a novel modified antisense oligonucleotide designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency. Nusinersen has been studied for safety, pharmacokinetics, and efficacy in both open-label and randomized controlled trials. The studies show improvement in motor function across SMA of all types. The most common adverse effects were respiratory tract infections, headache, back pain, constipation, and post-lumbar puncture syndrome. Relevance to Patient Care and Clinical Practice: Based on phase III trial data, nusinersen produced positive changes in the clinical course of patients with SMA. The acquisition and administration of nusinersen present a number of challenges in clinical practice. Its intrathecal delivery and costly price tag must be recognized. CONCLUSION: Nusinersen is safe and effective in patients with SMA. It was well tolerated across all studied age groups. SN - 1542-6270 UR - https://www.unboundmedicine.com/medline/citation/30008228/Nusinersen:_A_Treatment_for_Spinal_Muscular_Atrophy_ L2 - http://journals.sagepub.com/doi/full/10.1177/1060028018789956?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -