Phase 2 Trial of Amifampridine in SMA Type 3 Patients Fully Enrolled
A clinical trial testing oral amifampridine phosphate in people with spinal muscular atrophy (SMA) type 3 who are able to walk is…
Articles by Marisa Wexler, MS
Guidance on Zolgensma in Europe Notes Lack of ‘Evidence’ for Broad Use
Identifying those spinal muscular atrophy (SMA) patients who will benefit most from treatment with Zolgensma requires more than assessing traditional…
New Glia Cell Markers May Provide Insight Into Neuromuscular Diseases, Study Says
Perisynaptic Schwann cells — a specialized type of cell that helps facilitate communication between neurons and muscle cells…
Disparities Found in Parents, Children’s Perceptions of SMA
Perceptions about illness often don’t line up between children with spinal muscular atrophy (SMA) and their parents, with mothers…
Cure SMA Details Its ‘Clinical Trial Readiness Program’
A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by…
Interviews Probe How Unaffected Siblings Learn Genetic Implications of SMA
Siblings of people with spinal muscular atrophy (SMA) who do not have the disease themselves tend to learn about…
Too Little Known About Care Needs of Adults with SMA, Study Says
A better understanding of the needs of adults with spinal muscular atrophy (SMA), best ensuring for their mental and social…
Mom Writes of Family and Doctor ‘Partnership’ That Led to Baby’s Spinraza Treatment
A family’s journey through the diagnosis and treatment of their baby girl, born with spinal muscular atrophy type 1…
Taking Part in Adapted Sports Improves Mental Health, Study Finds
Among people with spinal muscular atrophy (SMA) and other neuromuscular disorders, participation in adapted sports is associated with greater…
Lack of Muscle Cell Signaling Seen to Kill Motor Neurons in SMA-LED2
Problems with the secretion of survival signals from muscle cells seems to be the dominant driver of motor neuron loss…