News

Isis Pharmaceuticals Receives $2.15M To Advance ISIS-SMN Rx In Children With SMA

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…

SMA Frontline Research Presented During 2015 SMA Researcher Meeting

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…

Cytokinetics Presents Positive Clinical Data For SMA Drug Candidate

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…

Isis Pharmaceuticals Reports Data On Spinal Muscular Atrophy

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 Plans to Award Nearly $2 Million in Research Grants

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…

BBrm02: BioBlast Pharma’s Novel Approach To Combat SMA

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,…

Cure SMA Publishes New Booklet on Clinical Trials

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 nonprofit organization…

Isis Pharmaceuticals Announces Results From Phase 2 Study of ISIS-SMNRx In Infants With SMA Type I

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,…

Cure SMA Releases Updates On SMA Drug Discovery Programs

Cure SMA, a non-profit organization focused on supporting research projects to find a cure for spinal muscular atrophy (SMA), has released an update on its drug pipeline of products currently under development to treat SMA. The organization has revealed its enthusiasm with both the increased number of projects…