News

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

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…

Long-term treatment with Spinraza (nusinersen) is safe and continues to promote better motor skills and respiratory function in children with spinal muscular atrophy (SMA), according to interim results from a Phase 3 clinical trial. This early data — from the open-label SHINE study — were in the presentation “Longer-term…

It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact, it rarely happens. But this year is different. Thailand’s Prince Mahidol Award Foundation has chosen the National Human Genome Research Institute — a division of the National Institutes of Health (NIH) —…

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…

A working group led by Cure SMA, and comprising 15 experts on spinal muscular atrophy (SMA), has developed new guidelines to help clinicians and caregivers decide when to administer therapy to infants with the disease. These come as a response to the recent recommendation by the Advisory Committee on…

Spinraza (nusinersen) improves motor and respiratory function in children with spinal muscular atrophy (SMA), according to results from different trials. Biogen will present the data at the American Academy of Neurology annual meeting, taking place April 21-27 in Los Angeles. SMA is a neurodegenerative disease caused by mutations in the SMN1 gene, which provides…

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…

Olesoxime (TRO19622) stabilizes the movement function of patients with Type 2 or Type 3 spinal muscular atrophy over the long term, an extension of a Phase 2 clinical trial shows. Roche will present the results at the 2018 annual meeting of the American Academy of…