Scholar Rock will start clinical trials of its spinal muscular atrophy therapy SRK-015 after studies in primates and mice showed that it helps improve muscle capacity and strength. The company will present the results of the preclinical-trial studies at the Cure SMA Annual Conference in Orlando, which started today and…
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Cytokinetics will present the latest developments on its drug candidate CK-2127107, also known as CK-107, for spinal muscular atrophy (SMA) during the Cure SMA 2017 Annual SMA Conference June 29 to July 2 in Orlando, Florida. Developed in collaboration with Astellas, CK-107 was designed to increase muscle function and…
Cure SMA announced the start of Phase 2 of its SMA Industry Collaboration, aiming to advance the development of specific goals. “The SMA Industry Collaboration is a multifaceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data,” according to a…
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…
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…
