AveXis recently reported favorable results from an ongoing Phase 1 clinical trial examining its candidate, AVXS-101, for the systematic delivery of gene therapy to young patients with spinal muscular atrophy (SMA). These preliminary results will help inform upcoming trials on gene therapy approaches. The open-label, dose-escalation study evaluating the safety and efficacy of…
RaNA Therapeutics recently released preclinical data on its gene upregulation technology, which selectively triggers gene expression within cells, as a treatment for spinal muscular atrophy (SMA). The data were given in a poster presentation at the recent Keystone Symposium for Chromatin and Epigenetics in British Columbia, Canada. SMA, a major cause of infant mortality, results from mutations or deletions…
The U.S. Senate Health, Education, Labor and Pensions (HELP) Committee recently passed an additional seven bills. Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families with support, observes that along with seven other bills…
FightSMA, an all-volunteer, parent-led nonprofit with a mission to accelerate research for a treatment for spinal muscular atrophy (SMA) by raising awareness and funding for SMA research, is marking its 25th anniversary with an international research conference April 7-9 in Alexandria, Virginia. The meeting will gather leading spinal…
This year’s SMA Researcher Meeting received a record number of abstract submissions, totaling 118 contributions from 14 countries, six different companies, and more than 50 institutions worldwide. The Researcher Meeting is expected to attract more than 300 researchers and clinicians from nearly 100 intuitions, plus other attendants, making this year’s meeting an…
Kavita Krishnaswamy, a PhD candidate in Computer Science at the University of Maryland, Baltimore County (UMBC), has launched an online survey on attitudes toward robotic aids and how they may improve the quality of life for those with physical disabilities, their family members and caregivers, and is inviting participation. Data…
Long-term exercise was found to benefit mice models of spinal muscular atrophy (SMA), providing important clues for designing rehabilitation programs in patients, according to a study titled “Long-term exercise-specific neuroprotection in spinal muscular atrophy-like mice” and published in the Journal of Physiology. SMA currently has no…
Medical education researcher Gregory Salinas, Ph.D. is conducting a survey to learn more about the challenges of patients suffering from type II or III spinal muscular atrophy (SMA). The 15-minute survey, mentioned in the website of Cure SMA, is for not only those who suffer from SMA but their caregivers, too,…
Cure SMA, an organization founded in 1984 to fund and invest in research for spinal muscular atrophy (SMA) treatments, also focuses on the day-to-day realities of patients and families, reaching about 4,000 SMA families every year through family support services with its 110,000-plus members and supporters. Among its other milestones, Cure SMA has invested more…
Texas Tech University researchers have identified a possible therapeutic target, the enzyme JNK3, for spinal muscular atrophy (SMA) that is independent of the causing genetic mutation. Their research, titled “Genetic Inhibition of JNK3 Ameliorates Spinal Muscular Atrophy,” was published in Human Molecular Genetics. SMA is a genetic neurodegenerative…
