News

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to AVXS-101, AveXis’ lead development candidate to treat spinal muscular atrophy (SMA) Type 1 in pediatric patients. AVXS-101 is a proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1, and, according to AveXis, is the…

In a study partly funded by Cure SMA, researchers from Ottawa Hospital Research Institute show that patterns of muscle breakdown in spinal muscular atrophy (SMA), occurring before any apparent neurodegeneration is taking place, differ depending on the severity of disease in mice. The report, “Differential induction…

Cure SMA has awrded a $50,000 Drug Discovery Grant to Brigham and Women’s Hospital researcher Kevin Hodgetts, PhD, at the Lab for Drug Discovery in Neurodegeneration for his project “Development of a Drug to Increase SMN2 Transcription.” The grant Dr. Kevin HodgettsPhoto Credit: Cure…

Diminished levels of the survival of motor neuron (SMN) protein causes spinal muscular atrophy (SMA), and also disrupts a process called endocytosis — crucial for the transfer of nerve signals between cells. The findings, published in the journal PNAS in the study, “Decreased function of survival motor neuron…

Cure SMA awarded a $50,000 drug discovery grant to researcher Barrington Burnett of Uniformed Services University of the Health Sciences for the project “Slowing SMN degradation to treat SMA,” which aims to characterize and validate a new survival motor neuron (SMN) protein modulator for the treatment of spinal muscular atrophy (SMA). Drug discovery…

U.S. Sens. Roger Wicker (R-Mich.) and Sherrod Brown (D-Ohio) – the lead sponsors of the National Pediatric Research Network Act (NPRNA) – are introducing a new piece of legislation that will require the National Institutes of Health (NIH) to implement the law, according to FightSMA. The NPRNA was signed into…

Progress reports on six treatment options for spinal muscular atrophy (SMA), all currently in clinical trials, were spotlighted at the recent 2016 Annual SMA Conference in Anaheim, CA. The updates, provided by study representatives, shows that efforts are advancing toward new therapies for SMA. Four of the six treatment programs — AVXS-101, nusinersen, RG7800/RG7916, and…

Researchers at the National Tsing Hua University in Taiwan discovered seven new genes affecting severity and time of onset of spinal muscular atrophy (SMA). The finding offers researchers clues for what molecules to target in new drug development attempts. The study, “An Integrative Transcriptomic Analysis for Identifying…

The Spanish National Research Council (CSIC) recently unveiled a prototype of what it believes is the world’s first assistive exoskeleton designed to enhance mobility for children with the degenerative illness spinal muscular atrophy (SMA), a rare disease that affects 1 in 10,000 babies born in Spain. The 26-pound device, made primarily of…

Cure SMA recently granted a $304,000 drug discovery award to Livio Pellizzoni, assistant professor of Pathology & Cell Biology at Columbia University for a new research project with potential to treat spinal muscular atrophy (SMA). Pellizzoni and collaborators at Northwestern University have identified an altered cellular pathway in SMA patients, which may…