Biogen Indec and Isis Pharmaceuticals Inc. recently announced they will start recruiting patients for NURTURE, a multi-center, Phase 2 clinical trial that will evaluate the efficacy of ISIS-SMNRx (ISIS 396443) in pre-symptomatic newborns that have a genetic diagnosis of Spinal Muscular Atrophy (SMA). The primary objective of the…
Determining the exact mutations responsible for spinal muscular atrophy (SMA) can be difficult. The condition is caused by complete deletion of the gene for survival motor neuron 1 (SMN1) in 95% of SMA patients. In the other 5%, only one copy of SMN1 is missing, and the other copy…
Dr. Martin Lemay A research project dedicated to the posture and breathing of patients who suffer from spinal muscular atrophy (SMA) has been awarded a $50,000 grant from Cure SMA. The grant awarded to Martin Lemay, PhD, is the last to be…
The Cure SMA has organization awarded a research grant totaling $80,000 to support a project from Linda Lowes, PT, PhD, at Nationwide Children’s Hospital. The project is entitled “Development of An Innovative Outcome Measure to Define Disease Progression in SMA Type I for Use in the Home or Clinic” and…
Perhaps the result of a gene duplication event 5 million years ago, humans have two copies of the gene for survival motor neuron (SMN). Patients with spinal muscular atrophy (SMA) lack SMN1 and have a missing piece of transcript from SMN2. Although SMN protein produced from SMN2 is shortened…
After working for 30 years funding research projects dedicated to spinal muscular atrophy (SMA) and facing the challenges related to it, Cure SMA is launching a new booklet to assist in the introduction of the research process. “Advancing…
The oil and gas company Newfield Exploration awarded a $2,500 grant to nonprofit organization Cure SMA. The funding will be invested in Cure SMA’s main mission of supporting both patients and families who suffer the burden of spinal muscular atrophy (SMA) as well as research projects working to…
Cure SMA has expanded its research program focused on finding treatments and a cure for spinal muscular atrophy (SMA) by providing an additional $315,000 in funding to support drug discovery at the California Institute of Biomedical Research (CALIBR). The grants aim to accelerate a research project…
Dallas, Texas-based AveXis, Inc., a biotechnology company currently developing an experimental therapy for Spinal Muscular Atrophy (SMA) through the use of innovative gene therapy, recently announced that they have established a European Union (EU) based wholly-owned subsidiary called AveXis EU, Ltd. This subsidiary will be used by AveXis to advance development of its…
“This is an exciting time for spinal muscular atrophy (SMA) patients, families and researchers,” indicated an article published in the journal F1000Prime Reports. “Not only are multiple clinical trials based on sound preclinical data completed, underway or planned, we also are rapidly gaining a better…
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