News

The non-profit organization Cure SMA has provided a $140,000 research grant to support the development of a project conducted by Chad Heatwole, MD, at the University of Rochester. The project entitled “Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials” is supported by the organization’s basic research…

The European Medicines Agency (EMA) has recognized a new European subsidiary of the biotech company AveXis, Inc. by granting it Small and Medium Sized Business Entities (SME) designation. AveXis EU Ltd. will work together with the parent company to improve the quality of life of patients who suffer…

A recent study published in the journal PLOS ONE revealed that a specific factor in the testis of spinal muscular atrophy (SMA) mice models can induce the expression of full-length SMN2 gene, potentially improving disease severity. The study is entitled “High Expression Level…

The non-profit organization Cure SMA is inviting its members and other interested parties to join the Annual SMA Conference, which is taking place between June 18 and 21 in Kansas City, Missouri. The event has been held by the organization since 1989 and it currently features the largest SMA-related research meeting…

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…