Novartis Poised for Phase 3b SMART Study of Zolgensma
Novartis will initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of the one-time gene…
Articles by Steve Bryson, PhD
Energy Problems in Muscle Cells of Type 3 Patients Evident in Exercise Study
Poorer oxygen update in muscle cells and greater fatigue was evident among ambulatory spinal muscular atrophy…
Scientists Reveal Architecture of SMN Complex, Altered in SMA
Scientists have revealed the structural architecture of the SMN complex, which is altered in people with spinal muscular…
Study Finds Varying Perspectives From Parents on Spinraza Therapy for Children
Parents of children with spinal muscular atrophy (SMA) reported different perspectives and concerns about Spinraza (nusinersen)…
Quantitative MRI Is Sensitive Measure of Muscle Decline
Young adults with spinal muscular atrophy (SMA) exhibited thigh muscle decline despite stable…
More Severe COVID-19 Symptoms Seen in Children with SMA Type 1
Among children with neuromuscular disorders, those with spinal muscular atrophy type 1 (SMA…
Informal SMA Caregivers Surveyed in Europe Report High Daily Burden
Non-professional caregivers who care for children with spinal muscular atrophy (SMA) carry a…
Financial Burden of SMA Much Higher Than Other Conditions, Analysis Shows
Direct medical costs associated with spinal muscular atrophy (SMA) are more than 50…
Measuring Neurological Impact of Polio Proves Useful in SMA, Study Reports
Researchers have found changes in the electrical activity between the brain and muscles in adults who had childhood…
Genetic Analysis of Families Finds Novel Mutations in SMA
Genetic analysis of 28 families with a member affected by spinal muscular atrophy…