Web Results
www.texaschildrens.org/about-us/news/releases/new-sma-treatment-topic-march-evenings-genetics

Mar 21, 2017 ... HOUSTON -- March 21, 2017 -- Spinal Muscular Atrophy (SMA) is a genetic disease that affects the nervous system and causes weakness of the ...

www.tandfonline.com/doi/full/10.1080/14656566.2019.1704732

Introduction: Spinal muscular atrophy (SMA) is one of the most common inherited neuromuscular disorders. It causes progressive muscle weakness and results ...

www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular-atrophy-gene-therapy.html

May 24, 2019 ... The FDA approved Novartis' Zolgensma, a one-time treatment for ... at the Healthy Returns conference in New York City on May 21, 2019.

journals.sagepub.com/doi/full/10.1177/2633105520973985

Nov 23, 2020 ... Spinal muscular atrophy (SMA) is one of the leading causes of infant ... South Plainfield, New Jersey and Hoffmann-La Roche, Basel, ...

www.forbes.com/sites/joshuacohen/2019/06/05/at-over-2-million-zolgensma-is-the-worlds-most-expensive-therapy-yet-relatively-cost-effective

Jun 5, 2019 ... Zolgensma can be a one-time, life-saving treatment that allows for ... As with any new treatment, there is invariably uncertainty about its ...

patienteducation.asgct.org/disease-treatments/sma

Sep 13, 2021 ... Because of this, it can be risky to avoid other forms of care or approved treatments while waiting for a new treatment approval. It is also ...

childrensnational.org/visit/conditions-and-treatments/genetic-disorders-and-birth-defects/spinal-muscular-atrophy

Learn about spinal muscular atrophy which is a genetic disease that affects ... treatment (approved December 2016) and new treatments in clinical trials.

www.nbcnews.com/health/health-news/2-1-million-drug-treat-rare-genetic-disease-approved-fda-n1009956

May 24, 2019 ... The drug, Zolgensma, is being called a "miracle drug" and will be used to treat children with spinal muscular atrophy, or SMA.

www.scripps.edu/newsandviews/e_20180516/SMA-genetic-disease.html

May 16, 2018 ... Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die ...

www.mdedge.com/neurology/article/219378/rare-diseases/spotlight-sma-part-2-spinal-muscular-atrophy-treatment

Mar 20, 2020 ... This supplement was developed in collaboration with Cure SMA. ... With newly available disease-modifying therapies, the phenotype of spinal ...

www.fiercepharma.com/pharma/novartis-touts-latest-zolgensma-data-as-sma-competition-against-biogen-roche-heats-up

Jun 18, 2021 ... As Roche's new spinal muscular atrophy drug, Evresdi, ... The results show that when patients were treated with Zolgensma before symptom ...