The U.K’s National Institute for Health and Care Excellence (NICE) has formally invited Biogen to submit Spinraza (nusinersen) for Single Technology Appraisal (STA) on its course toward possible NHS funding. Spinraza is the first approved spinal muscular atrophy (SMA) treatment in the E.U., the United States, and other…
Arm cycling training increased spinal muscular atrophy type 2 patients’ endurance and improved their results on movement tests, a small pilot study indicated. Despite researchers’ expectations, the training had no effect on patients’ levels of SMN protein or other factors that could help counter the disease. Nonetheless, the team at Hacettepe…
AveXis plans to launch clinical trials of its gene therapy AVXS-101 in a wider range of spinal muscular atrophy (SMA) patients — mirroring the company’s belief that the treatment may also benefit those with milder forms of the disease. The planned studies include one enrolling presymptomatic patients with…
Nearly 400 people have registered to attend Europe’s first-ever conference on spinal muscular atrophy, a rare neurological disease believed to affect between 21,000 and 25,000 people across the continent. The International Scientific Congress on Spinal Muscular Atrophy, set for Jan. 25-27 in the historic Polish city of Kraków, is…
RNA-processing proteins, including the FUS protein, may be involved in the underlying molecular mechanism that causes spinal muscular atrophy (SMA), a pilot study suggests. The study, “The expression of SMN1, MART3, GLE1 and FUS genes in spinal muscular atrophy,” was published in the journal Folia…
Cure SMA is granting $450,000 to help increase capacity at spinal muscular atrophy (SMA) sites across the United States. The money — with $50,000 to be given to nine sites — aim to boost the number of patients that sites can follow, treat and monitor. Five of the…
Researchers have shown that the technology called antisense oligonucleotides, used in the only FDA-approved therapy for SMA, Spinraza (nusinersen), can be improved upon. They designed two new sequences that significantly increased SMN production in human cells and in a mouse model of SMA when administered alone or combined. The…
Spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) was approved recently by the Korean Ministry of Food and Drug Safety, Biogen Korea announced. The treatment is the first (and only) SMA-specific therapy approved by the U.S. Food and Drug Administration (FDA) for both children and adults, based on…
The U.S. Food and Drug Administration has given AveXis a list of information it needs to consider approving AVXS-101 as a treatment for spinal muscular atrophy Type 1. Company officials met with regulators to discuss the results of a Phase 1 clinical trial (NCT02122952) of the gene therapy. They also discussed…
The biotech Scholar Rock has obtained $47 million in new financing to move its spinal muscular atrophy therapy SRK-015 into clinical trials during the first half of 2018. Invus led the financing round, which included Redmile Group and a number of other investors. “We are delighted by the strong…
