Cure SMA and our patient community celebrate the first approved drug for SMA

Gene Therapy
J GlascockJ Jarecki

Abstract

Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)-a disease affecting motor neurons, that robs patients of their ability to walk, eat and even breathe. Since 1984, we have directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. On 23 December, 2016, the Food and Drug Administration (FDA) announced approval of Spinraza, a treatment developed by Biogen and Ionis, making it the first-ever approved therapy for SMA. Cure SMA provided early research funding in 2003 leading to the discovery of ISS-N1 sequence, now targeted by Spinraza. We are pleased that our strategy of providing seed funding for research to either identify new therapeutic strategies or de-risk early stage ones, has proven successful with Spinraza's approval. The approval of Spinraza provides great hope to the SMA community and represents decades of hard work and perseverance by families, researchers, pharmaceutical companies and the FDA. Our hope is that Spinraza is the leading edge of a robust drug pipeline, and with our deep expertise in every aspect of SMA, we remain committed to do everything we can to support research and drug development to achieve the greatest possible eff...Continue Reading

References

May 26, 1999·Proceedings of the National Academy of Sciences of the United States of America·C L LorsonB Wirth
Mar 1, 2002·Muscle & Nerve·Mark B Bromberg, Kathryn J Swoboda
Jul 9, 2009·Nature Reviews. Neuroscience·Arthur H M Burghes, Christine E Beattie
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May 17, 2015·Seminars in Perinatology·Han C PhanRodney Howell
Feb 24, 2016·Annals of Clinical and Translational Neurology·Stephen J KolbUNKNOWN NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators
Dec 28, 2016·Annals of Neurology·Michelle A FarrarMatthew C Kiernan

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Citations

Nov 15, 2019·Muscle & Nerve·Bakri ElsheikhStephen J Kolb
May 21, 2019·Journal of Market Access & Health Policy·Daniel C MaloneOmar Dabbous
Dec 22, 2019·Stem Cell Reviews and Reports·Raffaella Adami, Daniele Bottai
Aug 8, 2019·Acta neurologica Belgica·Antoon Meylemans, Jan De Bleecker
Dec 31, 2019·Biotechnology Advances·Cui-Cui MaYu-Quan Wei

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