Patients with spinal muscular atrophy (SMA) are subdivided into four categories based on disease severity and age of diagnosis, with type I the most severe and diagnosed in infancy and type IV the least severe and diagnosed in adulthood. It is known that the copy number of SMN2 genes…
News
The nonprofit organization Cure SMA has announced the launch of a new chapter based in Virginia. The new Cure SMA Virginia Chapter will not only serve residents of the state, but also those with the disease and their families in Washington DC. The establishment of the Virginia chapter…
Researchers in the Division of Pharmacology, Department of Biochemistry and Molecular Biology at Kobe University Graduate School of Medicine in Japan are looking at ways of modeling spinal muscular atrophy (SMA) using cells derived from SMA patients. In a recent report published in Stem…
The non-profit organization Cure SMA is praising families that donate human tissue from their deceased relatives who suffered from spinal muscular atrophy (SMA), as the donations are crucial for a research program assessing the damage caused by SMA to the nervous systems of patients. The research is dedicated to increasing…
A study published in the Kobe Journal of Medical Sciences revealed a new method for the diagnosis of spinal muscular atrophy (SMA) by specifically detecting deletions in the SMN1 gene. The study was led by researchers at Kobe University Graduate School of Medicine and…
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…
