Web Results
www.npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever

May 24, 2019 ... Zolgensma, a new drug approved by the FDA Friday, costs more than $2.1 ... it appears it can save their lives with a one-time treatment.

www.bloomberg.com/news/features/2019-08-20/how-parents-get-zolgensma-the-world-s-most-expensive-drug

Aug 20, 2019 ... Gene therapy Zolgensma is the world's most expensive medicine. ... learning curve with a new treatment, particularly one this revolutionary, ...

www.m4rd.org/2020/03/31/spinal-muscular-atrophy

Mar 31, 2020 ... In June 2017, Nusinersen (SpinrazaTM) became Europe's first approved disease-modifying medication for the treatment of SMA.

stemcellthailand.org/therapies/spinal-muscular-atrophy-sma

NEW Neural Cell Treatment for Spinal Muscular Atrophy “SMA” Using Stem Cells. SMN1 SMN2 Plus Neurotrophic factor 2021 Protocol requires…

news.brown.edu/articles/2013/04/sma

Apr 9, 2013 ... ... at Brown University and Boston Children's Hospital have identified a new potential mechanism for developing an SMA treatment.

ufhealth.org/news/2019/uf-health-patient-first-receive-new-gene-therapy-neuromuscular-disease-fda-approval

Jun 13, 2019 ... It affects about 1 in 11,000 babies, according to Cure SMA. The U.S. Food and Drug Administration approved Zolgensma on May 24. It works by ...

www.ptcbio.com/our-pipeline/areas-of-interest/sma

SMA is a genetic disorder caused by the mutation or deletion of the survival of ... and develop new small-molecule therapeutics for use in the treatment or ...

www.keionline.org/charity-nih-funding-related-to-zolgensma

​​Zolgensma, the Novartis brand name for the new $2.1 million gene therapy for treatment of Spinal Muscular Atrophy (SMA), appears to be a remarkable ...

www.reuters.com/article/us-novartis-biogen-sma/biogen-sma-drug-price-novartis-estimates-for-its-treatment-far-too-high-u-s-group-idUSKCN1RF2L7

Apr 3, 2019 ... Biogen Inc should slash the price of its spinal muscular atrophy (SMA) ... The extremely high cost of new treatments for rare conditions has ...

onlinelibrary.wiley.com/doi/full/10.1111/dmcn.14027

Sep 17, 2018 ... In New Zealand, an expanded access programme is opened, and in Canada price negotiation for the treatment is in progress.

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 ...