AveXis is working with the U.S. Food and Drug Administration (FDA) to overcome final regulatory hurdles before starting a pivotal trial of AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA type 1) later in 2017. AVXS-101 is a gene therapy for SMA that has shown…
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In the United Kingdom, the SMA Trust is working to promote research and development of new treatments for spinal muscular atrophy (SMA). By providing funding and bringing together researchers, patients, healthcare professionals, and pharmaceutical industry representatives, it strives to accelerate research that might bring solutions to patients. In a…
A revved-up campaign to persuade the U.S. government to ask states to screen newborns for spinal muscular atrophy will be a hallmark of this year’s SMA Conference in Orlando. The agenda includes a symposium to train what the conference’s organizers call spinal muscular atrophy champions to push for newborn screening in their…
Anthem recently issued an updated insurance policy, expanding its coverage for the use of Spinraza (nusinersen), the first approved treatment for spinal muscular atrophy (SMA). The update revises the criteria for Spinraza in the treatment of onset of SMA-associated signs and symptoms to children younger than 21 months old. The previous…
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,…
Spinal muscular atrophy (SMA) patients in the European Union will soon have access to Spinraza (nusinersen), the first treatment approved for people with this disease and one able to treat almost all types of SMA. The European Commission announced late Thursday that it had approved Spinraza, marketed by…
Continuous simulation training for family caregivers of children with spinal muscular atrophy (SMA) leads to improved assistance at home and better clinical outcomes, according to new research. The study, “An Evaluation of a Continuing Education Program for Family Caregivers of Ventilator-Dependent Children with Spinal Muscular Atrophy (SMA),” appeared in the…
Alberto Kornblihtt, PhD, professor and group leader at the Universidad de Buenos Aires in Argentina, has been awarded a $140,000 grant for a project examining genetic changes that affect how much SMN protein is produced from the SMN2 gene. The study may greatly impact the spinal muscular atrophy (SMA) research…
Cure SMA has awarded a $75,000 basic research grant to Dr. Stephen J. Kolb of Ohio State University (OSU) for a project that seeks a deeper understanding of motor neuron pathology in spinal muscular atrophy (SMA). The project, “Arrested Development or Neurodegeneration? An approach to understand developmental motor neuron pathology…
The U.S. Department of Health and Human Services; Advisory Committee on Heritable Disorders in Newborns and Children will review spinal muscular atrophy (SMA) for inclusion in the agency’s Recommended Uniform Screening Panel (RUSP). The application, prepared and submitted by a newborn screening working group involving staff and members of Cure…
