Web Results
www.nhs.uk/conditions/spinal-muscular-atrophy-sma/treatment

It's not currently possible to cure spinal muscular atrophy (SMA), but research is ongoing to find new treatments. Treatment and support is available to ...

www.neurology.columbia.edu/patient-care/specialties/child-neurology/spinraza-treatment-spinal-muscular-atrophy-sma-patients

Spinraza (nusinersen) has been approved by the FDA and is now available for treatment of patients with SMA due to SMN1 mutations. The CUIMC - SMA Clinical ...

www.nice.org.uk/news/article/nice-approves-life-changing-gene-therapy-for-treating-spinal-muscular-atrophy

Mar 8, 2021 ... A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy (SMA) is set to become the ...

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 directly ...

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

May 31, 2019 ... Competition. Zolgensma is the second treatment approved for SMA, following Biogen's Spinraza. Now that there's competition in the marketplace, ...

www.ema.europa.eu/en/news/new-gene-therapy-treat-spinal-muscular-atrophy-corrected

Mar 27, 2020 ... EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma (onasemnogene ...

www.the-scientist.com/news-opinion/concerns-over-efficacy-and-cost-of-muscle-wasting-treatments-68144

Nov 11, 2020 ... In 2019, the FDA approved Zolgensma, a gene therapy that provides a functional copy via viral vector of the gene encoding SMN. The one-time ...

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.clinicaltrialsarena.com/projects/zolgensma

Zolgensma® (onasemnogene abeparvovec-xioi) is a gene replacement therapy indicated for the treatment of spinal muscular atrophy (SMA) in paediatric patients ...

www.theguardian.com/society/2021/mar/08/nhs-use-worlds-most-expensive-drug-treat-spinal-muscular-atrophy-zolgensma

Mar 7, 2021 ... Zolgensma, which costs £1.79m for one-off treatment, will be available ... “As is the case with many new treatments for very rare diseases, ...

www.fda.gov/news-events/press-announcements/fda-approves-oral-treatment-spinal-muscular-atrophy

Aug 7, 2020 ... The U.S. Food and Drug Administration today approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular ...