MicroRNAs Can Predict Response to Spinraza, Study Suggests
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 response to...
UK Collaboration Gets £1.25M Grant to Develop ‘Smart Suit’
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. The “SMART...
CADTH Panel Recommends Against Reimbursing Spinraza for Adults
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 independent,...
Evrysdi Safe in Type 1, 2 Patients in US Access Program Before Approval
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 analysis of...
Using Cupping During Physical Therapy For Patients With SMA
Recently, Michael, our patient with SMA, has exhibited some increased tenderness and reduced tissue mobility to his calf muscles bilaterally. This is no surprise since he doesn’t wear footrests and is constantly in a plantarflexed, or toe-down position. In this...
Zolgensma Safe in Children Heavier Than Those in Trials, Data Show
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 patients...
From Actor to Attorney, Woman’s ‘Work Ethic Outshines My Disability’
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 named to the...
Spinraza Stabilizes Motor Function for Adults With SMA, Study Reports
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 safety...
New Gene Therapy Shows Potential for Safety, Efficacy in Mouse Model
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 reported. The...
Parents of SMA Children Put Health as Top Priority at COVID-19’s Onset
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. The study,...
Disease Severity May Be Impacted by SMN Protein in Rare SMA Cases
The stability and self-interacting ability of SMN, the protein that is abnormally low in people with spinal muscular atrophy (SMA), may impact disease severity in rare cases, a small study suggested. Based on their findings, the researchers said new treatment...
Type 1 Children on Evrysdi Continue to Hit Milestones After 3 Years
Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of life are continuing to gain motor skills after three years of treatment, according to new data from the FIREFISH trial. “These long-term...