A team of researchers from the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) recently published a study that suggests fruit flies and brewer’s yeast can shed light on the root of Spinal Muscular Atrophy, today’s leading genetic cause of death among infants. This debilitating,…
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Isis Pharmaceuticals recently announced it received $2.15 million from Biogen to advance CHERISH, the ongoing pivotal Phase 3 study assessing ISIS-SMNRx in children suffering with spinal muscular atrophy (SMA). Isis has now received over $120 million from Biogen to advance this investigational therapy. CHERISH a Phase 3 ISIS-SMNRx study; it is a double-blinded, randomized, 15 month study in almost 120…
During the 2015 Cure SMA Researcher Meeting, a number of novel studies focusing on Spinal muscular atrophy (SMA) were presented under the topic “Emerging Trends in Motor Neuron Pathobiology.” SMA is neurodegenerative disease caused by a genetic defect in the Smn1 gene (short for spinal motor neuron-1). This alteration results in the loss of…
During the SMA Annual Conference Cytokinetics presented results from their Phase 1 study of CK-2127107, a novel small molecule activator of the fast skeletal muscle troponin complex to improve skeletal muscle function in diseases and medical conditions associated with neuromuscular dysfunction, muscular weakness, and/or muscle fatigue. The company, in collaboration with Astellas, recently announced…
Cure SMA, a non-profit organization dedicated to funding groundbreaking research on treatments for SMA, and providing families affected by this genetic disease to support they needs, is excited to welcome Dr. Mary Schroth, a medical professional education consultant, to their team. She will be working with Cure SMA a few…
Isis Pharmaceuticals, Inc. recently revealed its latest data on the Phase 2, open-label and multiple-dose study of ISIS-SMNRx in children diagnosed with spinal muscular atrophy (SMA) who are still receiving treatment in an open-label extension (OLE) study. Muscle function scores and motor function tests were preformed in children receiving ISIS-SMNRx treatment, and data proved consistent with previous…
Cure SMA, an organization committed to fund and direct comprehensive research that drives breakthroughs in treatment and care for SMA patients, has just announced plans to award $1.995 million worth of research grants for the upcoming months. These grants cover all 4 focused areas of Cure SMA’s research model: basic research, drug…
In a recent press release BioBlast Pharma Ltd., a clinical-stage orphan disease-focused biotechnology company, announced positive results of its preclinical proof-of-concept studies, conducted both in vitro and in vivo, for the company’s premier drug candidate BBrm02 to treat patients with Spinal Muscular Atrophy (SMA). BBrm02 belongs to a family of small molecule non-glycoside,…
An increasing amount of research on drugs to treat spinal muscular atrophy (SMA) is successfully progressing as a consequence of clinical trials, and Cure SMA believes there is a need to inform and educate the community about the clinical trial process. To do so, the…
Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2 trial of ISIS-SMNRx in infants with a diagnosis of spinal muscular atrophy (SMA) type I. The company had previously reported results from this trial which included assessments of muscle function, event-free…
