The initial position of England’s National Institute for Health and Care Excellence (NICE) is to recommend against adding Roche’s Evrysdi (risdiplam) to the list of medications available to spinal muscular atrophy (SMA) patients through the country’s national health service (NHS). Lack of evidence of Evrysdi’s efficacy in babies genetically diagnosed…
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…
Some people with disabilities say they’ve never wished for different circumstances. Well, I’m about to enter a black hole with you, in revelation of an honest confession: I’ve cast so many wishes into the night, I’ve nearly robbed the entire sky of stars. Odds are, you’ll see my face…
Evrysdi (risdiplam) has been approved by Health Canada for the at-home treatment of spinal muscular atrophy (SMA) in people ages 2 months and older. “The Health Canada approval of Evrysdi is a welcome addition in our ability to treat SMA. It is an efficacious treatment that significantly increases…
I’ve dreamed of cures. Miracles that occur in the blink of an eye. One minute, I’m sitting in my wheelchair, and the next, I’m standing, walking, running. I’m wobbly on my feet, of course, and crying, because everything has changed, and I’m probably overwhelmed by the newness of it all…
Evrysdi (risdiplam) continues to improve survival and to allow infants with spinal muscular atrophy (SMA) type 1 to achieve key motor milestones, according to two-year data from the second part of the FIREFISH trial. “It is encouraging to see that infants continued to improve after 12 months of treatment, with twice as many…
According to my mother, my columns are the first result when you Google “Evrysdi (risdiplam) denial.” Which isn’t surprising. I’ve written several scathing columns about the application process, from verifying my diagnosis to correcting blatantly false insurance claims. The squeaky wheel gets the grease, or so the…
The European Commission has approved Evrysdi (risdiplam) as the first oral and at-home treatment for adults, children, and infants 2 months and older with nearly all types of spinal muscular atrophy (SMA). Eligible patients include those with a clinical diagnosis of SMA type 1, 2, or 3 or carrying one to four…
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 continues to safely and effectively prevent motor function decline for at least five years, allowing the achievement…
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. Evrysdi (risdiplam) continues to improve or stabilize motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3,…