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…most common genetic cause of infant mortality. ISIS-SMNRx is an antisense drug designed to treat all types of SMA. (Logo: http://photos.prnewswire.com/prnh/20130807/LA60006LOGO) The preliminary data reported today is from a follow-up…

…celebrating the opening of the SMA (spinal muscular atrophy) Clinic, a center devoted to the treatment and management of a group of diseases that affect motor neurons of the spinal…

…to histones tends to open the local structure, promoting gene expression, while removing them makes it more compact, and less likely to be transcribed to mRNA. Histones are removed by…

…for patients with spinal muscular atrophy (SMA) Type 1 using the intravenous (IV) formulation produced by the company’s Good Manufacturing Practice (GMP) commercial manufacturing process. The company plans to initiate…

…in drug development, the experiences of patients taking Evrysdi, and the future of SMA therapeutics. Please see below for links to the articles. https://smanewstoday.com/news-posts/2020/08/10/lucky-genetics-and-hard-work-smas-leap-to-3-dmts-in-4-years/ https://smanewstoday.com/news-posts/2020/08/11/sma-foundation-little-toolbox-helped-pave-way-to-disease-modifying-therapies/ https://smanewstoday.com/news-posts/2020/08/12/evrysdi-has-parents-totally-optimistic-for-future-of-boy-with-sma-type-2/ https://smanewstoday.com/news-posts/2020/08/13/expertise-rna-biology-at-core-of-ptc-formative-work-with-evrysdi/ https://smanewstoday.com/news-posts/2020/08/14/sma-debate-might-systemic-treatment-be-best/ https://smanewstoday.com/news-posts/2020/08/17/everyday-good-using-evrysdi-risdiplam-61-year-old-with-sma-type-3-says-interview/ https://smanewstoday.com/news-posts/2020/08/18/combinations-muscle-therapies-likely-next-focus-for-sma-experts-say/…