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

Gene Therapy volume 24, pages 498–500 (2017)Cite this article

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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 effect for each and every SMA patient.

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Authors and Affiliations

  1. Cure SMA, Elk Grove Village, Illinois, USA

    J Glascock, M Lenz, K Hobby & J Jarecki

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  1. J Glascock
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  2. M Lenz
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  3. K Hobby
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Correspondence to J Jarecki.

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Competing interests

JG, ML, KH and JJ are employees of Cure SMA, and have no financial interest in this or any SMA drug program. Cure SMA has a financial interest in this and other SMA drug programs

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Glascock, J., Lenz, M., Hobby, K. et al. Cure SMA and our patient community celebrate the first approved drug for SMA. Gene Ther 24, 498–500 (2017). https://doi.org/10.1038/gt.2017.39

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