AveXis, Inc., a drug development company currently using gene therapy to address patients’ unmet needs suffering with orphan diseases such as Spinal Muscular Atrophy (SMA), has recently announced the completion of a $10 million financing set that was led by Deerfield Management and Roche Venture Fund. As part of the terms of…
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A recent review entitled “Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond,” recently published the journal Future Neurology, discussed current evidence on SMA with a specific focus on the existing treatment options. Spinal muscular atrophy (SMA) is an autosomal…
A study entitled “The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice” examined the effect of maternal diet in the gene SMN2 inducer in mice models with spinal muscular atrophy. The study was published in the journal Experimental Neurology. Proximal…
A recent review of current evidence on the pathogenesis of SMA based on the gene SMN was conducted by Kathryn Swoboda from the Department of Neurology, University of Utah School of Medicine. The perspectives Dr. Swoboda offers in the review suggest that experimental SMN-targeted therapeutics are indeed highly promising for treating…
South San Francisco based Cytokinetics, Incorporated and Astellas Pharma Inc. of Tokyo, Japan, have announced amendments expanding their research collaboration alliance for development and commercialization of skeletal muscle activators. The two companies have been cooperating on research and development efforts, the objective being to advance novel treatment therapies…
Spinal Muscular Atrophy (SMA) is a genetic disorder that causes progressive muscle wasting and robs the afflicted of physical strength and leads to mobility impairment due to its effect on motor nerve cells in the spinal cord. SMA eventually results in loss of the ability to walk, eat,…
Findings from a new study published in the journal Molecular Therapy offer a promising therapeutic strategy for patients with Spinal Muscular Atrophy (SMA). Spinal muscular atrophy (SMA) is an autosomal recessive disorder that causes loss of motor neurons and atrophy of the muscle, with patients presenting loss of…
In a recent study entitled “Spinal Muscular Atrophy Phenotype Is Ameliorated Either By SMN Increase Or Modulation Of Secondary Cell Death Events With RNA Therapy (S56.006),” a team of researchers from Milan, Italy, using a stem cell model sought to develop treatment approaches based on RNA for patients with…
A study entitled “Deletion of atrophy enhancing genes fails to ameliorate the phenotype in a mouse model of spinal muscular atrophy,” published in the journal Neuromuscular Disorders, found that removal of genes specifically implicated in muscle atrophy does not improve Spinal Muscular Atrophy phenotype. Spinal muscular atrophy…
Dallas-based biotechnology company AveXis, Inc. announced yesterday that they have successfully dosed the first patient in the intermediate cohort of their Gene Transfer Clinical Trial for chariSM A™ for Spinal Muscular Atrophy Type 1. The news comes just days after the company announced on December 5th that the dosing phase in the low-dose…
