Web Results
www.nytimes.com/2019/05/24/health/zolgensma-gene-therapy-drug.html

May 24, 2019 ... The therapy, to be sold as Zolgensma, alters the underlying genetic cause of spinal muscular atrophy and may permanently stop the disease. It is ...

my.clevelandclinic.org/health/diseases/14505-spinal-muscular-atrophy-sma

Apr 26, 2021 ... Going to a Cleveland Clinic location? New visitation guidelines · Masks required for patients and visitors (even if you're vaccinated) · 800.223&...

news.unchealthcare.org/2021/07/baby-first-at-unc-to-receive-gene-therapy-for-sma-thanks-to-early-check-newborn-screening

Jul 12, 2021 ... A Charlotte baby became the first at UNC Medical Center in Chapel Hill, N.C. to receive a breakthrough gene therapy treatment for spinal ...

www.hrsa.gov/sites/default/files/hrsa/advisory-committees/heritable-disorders/rusp/previous-nominations/sma-consumer-summary.pdf

Progress in screening and medical treatments can lead to new opportunities for newborn screening. To learn how a disorder is added to the RUSP, see Box A. What ...

stanfordhealthcare.org/medical-conditions/brain-and-nerves/spinal-muscular-atrophy.html

We are at the forefront of developing and validating new treatment approaches, such as Spinraza, the only treatment available for spinal muscular atrophy.

rarediseases.org/rare-diseases/spinal-muscular-atrophy

The following resource from Cure SMA provides a description of symptoms, as well as videos ... but the latest report described a girl with the same problem.

smanewstoday.com/sma-treatment

Aug 11, 2020 ... Zolgensma, a gene therapy developed by AveXis, a Novartis company, uses a genetically engineered virus to deliver a healthy copy of the SMN1 ...

www.wndu.com/2021/07/20/medical-moment-new-drug-sma

Jul 20, 2021 ... The FDA approved the drug last august after several clinical trials. In one trial, after 23 months or more of treatment, 81 percent of patients ...

investors.biogen.com/news-releases/news-release-details/biogen-advances-leading-research-spinal-muscular-atrophy-sma-new

Apr 19, 2021 ... Initial findings from the DEVOTE study suggest no new safety concerns and support ... Exploring Opportunities to Optimize Treatment in SMA

smauk.org.uk/blog/treatments-research

Biogen plans to initiate a new clinical trial called ASCEND, which will evaluate whether treatment with a higher dose of nusinersen has the potential to improve ...

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 ...