One way Cure SMA’s Annual SMA Conference encourages collaboration among academics, industry executives, government officials and families is through its Family Friendly Poster Session – a yearly event in which researchers present their findings about spinal muscular atrophy (SMA) in an accessible format. Registration is now open for the annual conference,…
Results from the first clinical trial of AVXS-101, a gene therapy for infants with spinal muscular atrophy (SMA) type 1, reveal dramatically improved survival as well as motor skills in most of the babies involved in the drug’s first clinical trial. Data presented by AveXis at the six-day AAN 2017 Annual Meeting — which…
Non-profit organizations for spinal muscular atrophy (SMA) and muscular dystrophy are reliant on help from people to raise both funds and awareness of the diseases. The money used goes towards research for better treatments, drugs, and understanding of the disease and to assist patients who have unique…
San Francisco-based Invitae has developed a genetic test to diagnose spinal muscular atrophy (SMA), the company announced at the March 21-25 American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting in Phoenix. SMA is the second leading genetic disorder affecting children in the United States. One in every 10,000 kids has SMA,…
Spinal muscular atrophy (SMA) is an inherited neurodegenerative disorder characterized by progressive muscle weakening and wasting due to the gradual loss of motor neurons, or nerve cells, that control muscles. Depending on the type of SMA, the age of onset and severity of the disorder…
Spinal muscular atrophy (SMA) is a rare genetic disease caused by the progressive loss of motor neurons, the specialized nerve cells that control voluntary movements, leading to muscle weakness and wasting. Due to the progressive nature of SMA, an earlier diagnosis is particularly important in terms of treatment: The…
The 2016 SMA Researcher Meeting, the largest gathering specifically dedicated to spinal muscular atrophy (SMA), set a record with 350 attendees who discussed cutting-edge research and the future of SMA patients’ needs. The goal of the annual event, held this year in Anaheim, California, is to create an open forum for the…
The 19th International SMA Researcher Meeting was recently held by Cure SMA in Kansas City, Missouri (June 18-20, 2015). Cure SMA announced that summaries from the 2015 meeting will be available showing the most relevant developments and findings presented at the meeting. SMA (spinal muscular atrophy) is a rare, devastating motor…
Two recent research projects involving studies into Spinal Muscular Atrophy are shedding new light on the deadly condition, which could in turn lead to viable next-generation therapies. The first new study, entitled, “SMA-causing missense mutations in survival motor neuron (Smn) display a wide range of phenotypes when modeled…
Consistent access to disease-modifying therapies (DMTs) is a major concern for parent caregivers of children with spinal muscular atrophy (SMA) in Canada, a study reported. Barriers to such treatments, as described by caregivers, included variable regulatory approvals across different Canadian provinces, high costs, and insufficient healthcare infrastructure. “Our…