In a recent webinar on Spinraza, the first approved treatment for spinal muscular atrophy (SMA), the patient advocacy group Cure SMA addressed issues such as access and insurance coverage, and answered questions from people affected by the disease. Cure SMA, a foundation that promotes SMA research and provides support for patients and their…
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Approximately one in six children and young people with spinal muscular atrophy (SMA) have abnormal sleep, according to a study published in the scientific journal Sleep Medicine. An additional one in six has an abnormal score on at least one sleep factor.
An analysis of U.S. orphan drug designations and approvals suggests that current incentives, detailed in the Orphan Drug Act (ODA) of 1983, have failed to stimulate the development of orphan drugs targeting spinal muscular atrophy (SMA) and other rare diseases. Rather, researchers at the University of Massachusetts-Amherst suggest that the…
Researchers may finally have figured out why a loss in the SMN protein that is crucial for all cells so quickly affects motor neurons, leading infants to develop spinal muscular atrophy (SMA). SMN aids in the transport of certain molecules in all cells, processing RNA, and the mutation-caused drop in its levels within cells…
Access to Spinraza (nusinersen), the first ever approved therapy for the treatment of spinal muscular atrophy (SMA), will be discussed in a webinar tomorrow.
Carl Rooney, a father of two from the English town of St. Ives, aims to run 2,017 miles to raise money for SMA Support UK, the charity that supported his brother, who has spinal muscular atrophy (SMA). The 39-year-old chose this marathon challenge hoping to give something back to the community,…
The patent application for gene therapy candidate INT41, developed by Vybion for the treatment of Huntington’s disease, spinal muscular atrophy (SMA), and other neurodegenerative diseases, was granted Track One status from the U.S. Patent and Trademark Office (USPTO). The patent application covers the treatment’s composition and methods of use.
Lowering levels of a newly identified protein, in combination with a splice-modifying therapy, improved symptoms and survival in animal models of spinal muscular atrophy (SMA), according to a recent study. The finding suggests that targeting the factor — called neuronal calcium sensor neurocalcin delta (NCALD) — could be beneficial for SMA patients,…
Vivonoetics is partnering with Great Lakes NeuroTechnologies to provide advanced respiratory assessment to the Roche FIREFISH Phase 2 clinical trial in infants with type 1 spinal muscular atrophy type 1 (SMA-1). Vivonoetics is the developer of VivoSense data analysis and software and wearable monitoring devices that have supported research…
RTI International has developed a groundbreaking program of free genetic-disease testing for newborns, with the initial screenings being for the spinal muscular atrophy (SMA) and fragile X syndrome. Although most American newborns are tested for a number of genetic diseases, screenings are not done for others because they are too…
