News Archives - SMA News Today The Web's Daily Resource for Spinal Muscular Atrophy News
- MicroRNAs Can Predict Response to Spinraza, Study Suggestsby Steve Bryson, PhD on May 27, 2022
MicroRNAs isolated from people with spinal muscular atrophy (SMA) types 2 and 3 before treatment predicted eventual responses to Spinraza (nusinersen) therapy, a study suggested. The study, “Muscle microRNAs in the cerebrospinal fluid predict clinical […]
- UK Collaboration Gets £1.25M Grant to Develop ‘Smart Suit’by Mary Chapman on May 25, 2022
Development of an exoskeleton suit to help those living with progressive neuromuscular conditions that affect their upper-body movement will advance due to a £1.25 million (about $1.5 million) grant from the People’s Postcard Lottery in the United Kingdom. […]
- CADTH Panel Recommends Against Reimbursing Spinraza for Adultsby Marisa Wexler, MS on May 23, 2022
A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with spinal muscular atrophy (SMA). CADTH is an […]
- Evrysdi Safe in Type 1, 2 Patients in US Access Program Before Approvalby Marta Figueiredo, PhD on May 20, 2022
Evrysdi (risdiplam) was generally safe and well tolerated in a broader group of children and adults with spinal muscular atrophy (SMA) types 1 and 2 than that included in clinical trials supporting the therapy’s approval. These were the findings of an […]
- Zolgensma Safe in Children Heavier Than Those in Trials, Data Showby Marta Figueiredo, PhD on May 18, 2022
Zolgensma appears generally safe and well tolerated among spinal muscular atrophy (SMA) patients weighing 8.5 kilograms (about 18.7 lbs) or more, with no new safety concerns identified in the heavier group. Notably, 8.5 kilograms was the maximum weight of SMA […]
- From Actor to Attorney, Woman’s ‘Work Ethic Outshines My Disability’by Hawken Miller on May 16, 2022
From being a child actor to a lawyer in the entertainment industry, Alexa Dectis, who has relied on a power wheelchair to get around since she was 2, has gone far. Dectis, diagnosed with spinal muscular atrophy (SMA) type 2 when she was 18 months old, was […]
- Spinraza Stabilizes Motor Function for Adults With SMA, Study Reportsby Marisa Wexler, MS on May 13, 2022
Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy (SMA) types 2 or 3, according to a study from Israel. The study, “Longer-term follow-up of nusinersen efficacy and […]
- New Gene Therapy Shows Potential for Safety, Efficacy in Mouse Modelby Lindsey Shapiro, PhD on May 11, 2022
A second-generation investigational gene therapy for spinal muscular atrophy (SMA) showed greater safety and efficacy than an earlier version, and it may be more effective than the currently approved gene therapy for SMA, a study in a disease mouse model […]
- Parents of SMA Children Put Health as Top Priority at COVID-19’s Onsetby Marisa Wexler, MS on May 9, 2022
For parents, protecting the health and well-being of a child with spinal muscular atrophy (SMA) was a top priority when the COVID-19 pandemic first hit the Netherlands in early 2020, despite the difficulties isolation can bring, an interview study reports. […]
- Disease Severity May Be Affected by SMN Protein in Rare SMA Casesby Steve Bryson, PhD on May 6, 2022
The stability and self-interacting ability of SMN, the protein that is abnormally low in people with spinal muscular atrophy (SMA), may affect disease severity in rare cases, a small study suggested. Based on their findings, the researchers said new treatment […]
