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AndroScience Corporation Awarded a $3.8 Million, 3-Year Milestone-Driven, Cooperative Translational Research Grant from the NIH to Develop an Oral Treatment for Spinal Bulbar Muscular Atrophy (Kennedy's Disease)
Date:1/25/2011

studies.  To date, ASC has provided robust proof of concept data using an ARD enhancer compound; demonstrating treatment ameliorates cardinal features of SBMA neuromuscular pathology, restores functional activity, and improves survival in a SBMA transgenic mouse model.

About Spinal Bulbar Muscular Atrophy (SBMA) – Kennedy's DiseaseSpinal and bulbar muscular atrophy (SBMA or Kennedy's Disease) is a rare hereditary neurodegenerative disease that affects lower motor neurons, with progressive muscle atrophy and weakness of the bulbar, facial, and limb muscles. The disease results in progressive dysphasia, motor dysfunction, and typically affects men in the fourth or fifth decade of life.  SBMA is caused by a mutation in the X-chromosome linked androgen receptor (AR) gene that results in excessive repeats of the amino acid glutamine (polyQ) within AR protein.  A molecular genetic test is clinically available which has a 100% mutation detection rate to definitively diagnose SBMA.  Neurotoxicity caused by these expanded polyQ AR aggregates is believed to play a pivotal role in the pathogenesis of SBMA.  SBMA is considered an orphan drug indication and no approved therapy exists.  Furthermore, very few drug candidates are being evaluated in clinical trials.  

About AndroScience CorporationAndroScience (ASC) is a privately held San Diego, California based pharmaceutical company applying expertise in natural product chemistry to develop proprietary small molecule drug compounds targeting disease dependent intracellular signaling events related to the Androgen Receptor (AR) and selective Signal Transducer and Activation of Transcription (STAT) pathways.  The company is focused on therapeutic indications of significant medical and commercial importance, including androgen-dependent diseases in both topical and systemic treatment settings and novel approaches to the treatment of cancer.  Active ARD enhancer
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