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…
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…
Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future — an initiative designed to expand the company’s drug pipeline and advance its muscle biology-directed drug candidates toward late-stage development and marketing strategies. The biopharmaceutical company specializes in first-in-class muscle activators for people with impaired muscle function, including spinal muscular atrophy (SMA) patients. The…
For the first time, scientists have shown that insufficient blood supply is likely to contribute to motor neuron loss in spinal muscular atrophy (SMA), a finding that potentially opens a new avenue into disease research. The study, entitled “Vascular defects and spinal cord hypoxia in spinal muscular atrophy,” was…
The non-profit organization Cure SMA has published the video and transcription of an event recently organized, titled “Clinical Regulatory Webinar.” On Monday, November 9th, Cure SMA hosted a one-hour webinar to discuss clinical trial design, expanded access, New Drug Application (NDA) processes, among other topics. Jill Jarecki, the research director at Cure SMA, was…
Members of Cure SMA‘s Medical Advisory Council (MAC) recently met for two days in Chicago to set the agenda for the care of SMA individuals and families. Mary Schroth, MD, a leading pulmonologist and professor of pediatrics, chairs the MAC, one of the most respected groups of SMA medical and clinical specialists in…
Cure SMA has been given 10 special tomato feeder seats by the Jadon’s Hope Foundation. The donation is meant to help the organization in its goal of not only finding a cure for spinal muscular atrophy (SMA) but also improving the quality of life for patients with this severe condition. The adaptive, comfortable and…
AveXis, Inc. has recently presented results from its ongoing Phase 1/2 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1 during the International Congress of the World Muscle Society in Brighton, United Kingdom. Lead investigator Dr. Jerry R. Mendell, director of the Center for Gene…
Genentech, a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions, recently announced an update on the clinical development of RG7800, its investigational SMN2 splicing modifier that is being developed for the treatment of patients with spinal muscular atrophy…
In a recent study published in the journal Stem Cells Translational Medicine, a team of researchers from the Cedars-Sinai Board of Governors Regenerative Medicine Institute have employed a gene-editing method that uses low-dose irradiation to edit the genome of patients’ stem cells. According to the study results, this strategy…
