José Lopes, PhD,  managing science editor—

The first patient has been dosed in the Phase 3 clinical trial of AveXis’ gene therapy candidate AVXS-101 in presymptomatic infants with spinal muscular atrophy (SMA) types 1, 2 and 3, the company announced. SPR1NT (NCT03505099) is an open-label, global study evaluating a one-time, intravenous AVXS-101 treatment in…

A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called  RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, early results of a two-part clinical trial show. Data from the study, “RG7916 significantly increases SMN Protein in…

Type 1 spinal muscular atrophy (SMA) babies treated with AveXis’ gene therapy candidate AVXS-101 continue to achieve unprecedented motor milestones and do not require breathing or nutritional support, according to recent data from the company. AveXis presented updated results from Phase 3 and Phase 1 trials…

Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will share its clinical results at the 2018 American Academy of Neurology (AAN) Annual Meeting, April 21-27 in Los…

Blood levels of a waste product from muscle metabolism could be used to see how spinal and bulbar muscular atrophy (SBMA) develops before symptoms appear, a Japanese study reports. The research on the waste product, creatinine, appeared in the journal Neurology. The title of the…

Spinal muscular atrophy (SMA) expert Basil T. Darras, MD, is optimistic about treatment advances and the possibility of a future cure, according to a recent interview in which he shared his thoughts on current SMA therapeutic approaches and care. Darras is a professor of neurology (pediatrics) at Harvard Medical School…

SRK-015 has received orphan drug status designation by the U.S. Food and Drug Administration (FDA) to treat muscle atrophy in patients with spinal muscular atrophy (SMA). SRK-105 is Scholar Rock’s lead product candidate, intended to improve muscle strength…

AveXis’ expects to start a Phase 3 clinical trial of its spinal muscular atrophy (SMA) gene therapy AVXS-101 in Europe in May. The STR1VE-EU trial (NCT03461289) is expected to include up to 30 children under 6  months of age with type 1 SMA, the disease‘s most severe and…

All 25 pre-symptomatic infants treated with Biogen’s Spinraza (nusinersen) in a Phase 2 clinical trial of spinal muscular atrophy (SMA) did not require permanent ventilation and exhibited motor improvements, according to a preliminary analysis. The ongoing NURTURE study (NCT02386553) is assessing the effectiveness and safety of treatment…

A disruption between two molecules that transport substances to the remote ends of special nerve fibers may result in neurodegenerative disorders such as spinal muscular atrophy (SMA) or amyotrophic lateral sclerosis (ALS), a study suggests. The study, “hnRNP R and its main interactor, the noncoding RNA 7SK, coregulate…