SadBaby, an organization that raises donations to support children with spinal muscular atrophy (SMA), is launching a crowdfunding platform. The project, based on digital currency donations, intends to gather funds from supporters and investors to help families financially with the costly treatments associated with SMA. “SadBaby is doing…

The UK SMA Newborn Screening Alliance is calling on people to take action and sign a petition requesting that spinal muscular atrophy (SMA) to be added to the U.K.’s National Health System (NHS) newborn screening program and funded. U.K. citizens and residents can sign the petition, which is collecting…

Evrysdi (risdiplam) has been approved in Japan as an oral, at-home treatment of spinal muscular atrophy (SMA). The flavored liquid therapy is also approved in the U.S., Europe, and Canada to treat all types of SMA in patients ages 2 months and older, and its use was favored by regulators…

Newborn screening (NBS) for spinal muscular atrophy (SMA) in Massachusetts allowed for early detection and treatment of affected infants, with most referred to a specialist within the first week of life, three-year data from the state’s SMA screening program show. Notably, the program used a tiered algorithm with screenings that…

Evrysdi (risdiplam) continues to be linked to motor function improvements in a broad range of people with spinal muscular atrophy (SMA), according to recent data from two ongoing Phase 2 clinical trials. These data were presented at the 2021 Virtual SMA Research & Clinical Care Meeting. Presymptomatic…

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…