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kidshealth.org/en/parents/sma.html

Spinal muscular atrophy (SMA) is a condition that causes muscle weakness and atrophy. There's no cure, but therapy and other treatments can help most people ...

www.mda.org/disease/spinal-muscular-atrophy

On Dec. 23, 2016, the U.S. Food and Drug Administration (FDA) approved Spinraza (nusinersen) for the treatment of SMA. Spinraza is designed to treat the ...

smanewstoday.com/sma-treatment

Aug 11, 2020 ... Spinraza (nusinersen), by Biogen, was the first disease-modifying therapy for SMA that the FDA approved in December 2016 to treat SMA in ...

www.statnews.com/2019/05/31/spinal-muscular-atrophy-zolgensma-price-critics

May 31, 2019 ... The Food and Drug Administration's decision was a pivotal day for those of us in the SMA community. Zolgensma, approved for children under 2, is ...

www.nature.com/articles/s41434-021-00273-7

Jul 19, 2021 ... Onasemnogene abeparvovec (Zolgensma®) is a novel gene therapy medicine, FDA-approved in May 2019 for the treatment of SMA.

www.managedhealthcareexecutive.com/view/an-overview-of-the-sma-treatment-landscape

Sep 27, 2021 ... The FDA has approved three targeted therapies for spinal muscular ... to treat children born with spinal muscular atrophy (SMA), a rare, ...

www.medpagetoday.com/neurology/generalneurology/94891

Oct 6, 2021 ... Gene replacement therapy with onasemnogene abeparvovec (Zolgensma) enabled symptomatic infants with spinal muscular atrophy type 1 (SMA1) to ...

www.uptodate.com/contents/spinal-muscular-atrophy

Oct 19, 2021 ... Zolgensma - one-time gene therapy for spinal muscular atrophy. Med Lett Drugs Ther 2019; 61:113. FDA approves oral treatment for spinal muscular ...

emedicine.medscape.com/article/1181436-treatment

Aug 11, 2020 ... In December 2016, the FDA approved nusinersen (Spinraza), the first drug approved to treat children (including newborns) and adults with SMA.

www.novartis.com/news/media-releases/new-zolgensma-data-demonstrate-age-appropriate-development-when-used-early-real-world-benefit-older-children-and-durability-5-years-post-treatment

Mar 15, 2021 ... Zolgensma® (onasemnogene abeparvovec) is the only gene therapy for spinal muscular atrophy (SMA) and the only SMA treatment designed to ...

www.childrenshospital.org/conditions-and-treatments/conditions/s/spinal-muscular-atrophy-sma/diagnosis-and-treatment

As yet, there is no complete cure for SMA. However, the discovery of the genetic cause of SMA has led to the development of several treatment options that ...