Many U.S. pediatricians fail to use developmental screening tools and are not familiar with the diagnostic requirements for spinal muscular atrophy (SMA), a study drawing on results of two surveys conducted by Cure SMA reported. These findings, from responses given by hundreds of pediatricians, support a continuing need to…

Blood levels of a stress-induced protein are significantly higher in young spinal muscular atrophy (SMA) patients and associate with levels of a nerve cell damage biomarker, a study shows. These findings from untreated SMA patients suggest that this protein, called heat shock 70kDa protein 7 (HSP70B), may be a…

The number of copies of the “backup” SMN2 gene and a rare genetic variant in this gene influence disease severity in people with spinal muscular atrophy (SMA), according to data from more than 2,000 people involved in a free SMA genetic testing program. Launched in 2018 by…

Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Zolgensma safely and effectively halts disease progression in pre-symptomatic spinal muscular atrophy (SMA) infants, according to updated…

Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference.   Zolgensma — given alone, after, or in combination with another spinal muscular atrophy (SMA) disease-modifying therapy…

I’m a groundhog. Well, symbolically, I’m a groundhog. I emerged from my burrow on Groundhog Day, 1986.  I’m reminded of this each year my birthday rolls around, when my mom recollects her story about being in labor with me as the hourglass sands of Feb. 1 dwindled, and I…

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal muscular atrophy (SMA) throughout 2020. We look forward to reporting more news to patients, family members, and caregivers dealing with SMA during 2021. Here are the top 10 most-read articles…

Direct medical costs associated with spinal muscular atrophy (SMA) are more than 50 times higher than those of matched control patients without SMA, according to a healthcare claims analysis. The primary driver of cost for infantile SMA cases was inpatient expenses,…

Novartis’ gene therapy Zolgensma is generally safe and effective in promoting motor improvements in children with spinal muscular atrophy (SMA) treated up to age 2, according to a multicenter study. However, “if the children are older and heavier, there is a potential for more liver injury [a known side effect…