Mutations in TBK1 Gene May Cause Rare Form of SMA: Case Study
A man in Brazil was found to have an exceptionally rare form of spinal muscular atrophy (SMA) — called…
Articles by Marisa Wexler, MS
New Technique Found Suitable for Body Composition Measures in SMA
A technique called bioelectrical impedance analysis (BIA) can be used to measure body composition in people with type 2 or…
More Than 1,200 People with SMA Live in Italy, Study Shows
There are more than 1,200 people with spinal muscular atrophy (SMA) living in Italy, and the vast majority are…
Patients With Less Muscle Loss More Likely to Respond to Spinraza: Study
People with spinal muscular atrophy (SMA) types 2 or 3 who start treatment with Spinraza (nusinersen) and show…
Costs of SMA ‘Extremely High’ Prior to Disease-modifying Treatments
Without disease-modifying treatment (DMT), spinal muscular atrophy (SMA) carries high mortality rates and steep healthcare costs, a study…
Antibody-ASO Conjugate System Improves Survival in SMA Mice
A new system that contains a gene-modulating therapy attached to an antibody that enables crossing into the nervous system improved…
Inflammatory Protein IL-8 May Predict Response to Spinraza: Study
Children with spinal muscular atrophy (SMA) who have lower levels of an inflammatory protein called interleukin-8 (IL-8) in the…
Babies’ Motor Function Improves After 2 Years on Evrysdi
After two years of treatment with Evrysdi (risdiplam), most babies with spinal muscular atrophy (SMA) type 1 in…
Apitegromab Showing Potential to Ease Fatigue, Aid Life Quality
Continued treatment with apitegromab, designed to strengthen muscles, led to stabilization or improvement in measures of fatigue and life…
SMA Patients, Caregivers Surveyed About Switching to Evrysdi
Children with spinal muscular atrophy (SMA) switching from Spinraza (nusinersen) to Evrysdi (risdiplam) are generally optimistic about…