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 lead…
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“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…
A team of Chinese researchers published their findings in the journal BMC Musculoskeletal Disorders where they show that the copy number of genes related to spinal muscular atrophy (SMA) differs between individuals and influences disease severity. The study is entitled “Molecular characterization and copy…
Specific gene mutations can help shed light on the possible causes of diseases such as spinal muscular atrophy (SMA). Several genes have already been associated with the disorder. SMA is a genetically-determined neuromuscular disorder characterized by loss of specific spinal cord neurons, which causes weakness of the limbs and muscle degeneration. This leads…
In an exclusive interview, SMA News Today Senior Columnist Maureen Newman spoke with Dr. Adrian Krainer of Cold Spring Harbor Laboratory. In conjunction with Isis Pharmaceuticals, Dr. Krainer and his team are making promising strides into better understanding how SMA works. In January, Dr. Adrian Krainer of Cold Spring Harbor Laboratory…
The first report of co-existing spinal muscular atrophy (SMA) with diabetes mellitus type 1 appeared in the journal Acta Biochemica Polonica, February 12, 2015. The report, titled “Coexistence of type 1 diabetes mellitus and spinal muscular atrophy in an 8-year-old girl: a case report,” came from…
Cure SMA, which supports research to find a treatment and an eventual cure for spinal muscular atrophy (SMA), has awarded a $140,000 grant to the research project “mTOR and Protein Synthesis in SMA” currently being conducted by Mustafa Sahin, MD, PhD, at Boston Children’s Hospital. The…
A collaborative international study published in the renowned journal Science revealed that specific small-molecule compounds are effective in preventing spinal muscular atrophy (SMA) progression in mouse models. The study is entitled “SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular…
To better understand spinal muscular atrophy (SMA), scientists must conduct studies using more than just cells in a culture dish. It is best to recapitulate the human body using a large animal model, but many scientists use mice due to their availability. To fill the gap of large animal…
A study published in the journal PLoS One revealed that members from specific protein families could be potential new targets for spinal muscular atrophy (SMA) therapies. The study is entitled, “Targeting SR Proteins Improves SMN Expression in Spinal Muscular Atrophy Cells.” SMA is…
