A lack of familiarity with spinal muscular atrophy (SMA) type 1 among primary health care staff often makes caring for infants with the disease a stressful experience for families, finds a study that explored how parents perceived various care settings, including emergency, hospitals and clinical care. The study, “The Experience…
The national nonprofit spinal muscular atrophy (SMA) support and advocacy organization Cure SMA has launched a grassroots campaign to have required newborn-screening for SMA in every state. The campaign was announced at the 2017 Annual SMA Conference held June 29-July 2 in Orlando, Florida. It was attended by…
Cytokinetics revealed preliminary data on the characteristics of patients with spinal muscular atrophy (SMA) screened for a Phase 2 trial to investigate the safety, tolerability and pharmacokinetics of CK-2127107. Data were presented by Cytokinetics’ director of clinical research, Stacy Rudnicki, MD, at the Cure SMA 2017 Annual SMA Conference in Orlando,…
Preliminary results from a phase 2 trial confirm that the investigational drug RG7916 targets the underlying genetic cause of type 2 and type 3 spinal muscular atrophy (SMA), PTC Therapeutics has announced. RG7916 is an oral drug that modulates the splicing of the SMN2 gene to increase production…
Spinraza (nusinersen) was just approved by Health Canada for the treatment of 5q spinal muscular atrophy (SMA) patients, Biogen Canada announced. This is the first therapy approved by Health Canada for the treatment of SMA. The drug is specifically indicated to treat 5q SMA, which represents about…
Infants with Spinal Muscular Atrophy (SMA) who do not yet show symptoms experience numerous benefits from Spinraza (nusinersen) treatment, according to data that Biogen will present at the Cure SMA 2017 Annual Conference in Orlando, Florida on June 29 to July 2. Among the host of presentations that the company…
Scholar Rock will start clinical trials of its spinal muscular atrophy therapy SRK-015 after studies in primates and mice showed that it helps improve muscle capacity and strength. The company will present the results of the preclinical-trial studies at the Cure SMA Annual Conference in Orlando, which started today and…
Cytokinetics will present the latest developments on its drug candidate CK-2127107, also known as CK-107, for spinal muscular atrophy (SMA) during the Cure SMA 2017 Annual SMA Conference June 29 to July 2 in Orlando, Florida. Developed in collaboration with Astellas, CK-107 was designed to increase muscle function and…
Cure SMA announced the start of Phase 2 of its SMA Industry Collaboration, aiming to advance the development of specific goals. “The SMA Industry Collaboration is a multifaceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data,” according to a…
AveXis is working with the U.S. Food and Drug Administration (FDA) to overcome final regulatory hurdles before starting a pivotal trial of AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA type 1) later in 2017. AVXS-101 is a gene therapy for SMA that has shown…
