Researchers at the Harvard Stem Cell Institute have identified a compound that can restore the SMN motor-neuron protein whose shortage is associated with spinal muscular atrophy (SMA). “This discovery opens up new lines of drug interrogation,” Lee Rubin, the senior author of the study, said in a news story written by Harvard’s Hannah…
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Genentech is working on the design of a Phase 3 clinical trial of olesoxime to address U.S. and European regulators’ concerns about the benefits versus the risks of the spinal muscular atrophy (SMA) therapy. Meanwhile, two Phase 2 trials of another Genentech SMA treatment, RG7916, are under way. The U.S.
The Neuromuscular Program at Stanford and Lucile Packard Children’s Hospital (LPCH) will hold the 3rd Annual Spinal Muscular Atrophy Educational Conference at California’s Stanford School of Medicine on Saturday, May 13. The conference, designed for spinal muscular atrophy (SMA) patients and caregivers, will take place from 10 a.m. to 3:30 p.m.
A meeting that the spinal muscular atrophy (SMA) community is having with Food and Drug Administration officials in the Washington area next week is full, but will be live-streamed. The gathering on April 18 in Hyattsville, Maryland, will focus on the development of therapies for SMA. Patients, their families and caregivers…
A recent survey showed that spinal muscular atrophy (SMA) patients and their families are generally supportive of genetic screening program for early detection of SMA. However, they still have a number of fears about the real outcome of the tests. These findings stressed the need of improved relations between those…
AVXS-101 may become a valuable gene therapy option for babies with spinal muscular atrophy (SMA) type 1, according to the results of a Phase 1 clinical trial conducted by AveXis. SMA is caused by mutations in the SMN gene, which leads to the impairment of motor neurons, which work to…
Dr. Jocelyn Côté, an RNA metabolism expert at the University of Ottawa, has received $140,000 in basic research funding from Cure SMA and Families of SMA Canada. Côté will investigate survival motor neuron (SMN) protein regulation for clues that could lead to new treatment approaches for spinal muscular atrophy (SMA).
Cytokinetics has begun enrolling a second group of patients for a Phase 2 clinical trial of CK-2127107 as a treatment for spinal muscular atrophy (SMA). The announcement follows an independent Data Monitoring Committee’s review of the therapy’s safety, absorption, distribution, metabolism, and excretion in Cohort 1. The randomized, double-blind, placebo-controlled,…
San Francisco-based Invitae has developed a genetic test to diagnose spinal muscular atrophy (SMA), the company announced at the March 21-25 American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting in Phoenix. SMA is the second leading genetic disorder affecting children in the United States. One in every 10,000 kids has SMA,…
Cytokinetics’ next-generation fast skeletal troponin activator CK-2127107 improves muscle function in mouse models of spinal muscular atrophy (SMA), according to a preclinical trial study. The findings were presented at the MDA Scientific Conference in Arlington, Virginia. Cytokinetics and Astellas Pharma are developing CK-2127107 (CK-107) as a treatment for SMA and…
