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Registered: 12-08-2010
Posts: 10
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Has anyone heard of this?

Cabaletta for (SBMA – Kennedy’s disease)

SBMA, also known as Kennedy’s disease, is characterized by the degeneration and loss of lower motor neurons in the brainstem and spinal cord. Patients present with weakness in muscle function, including severe difficulty in swallowing and aspiration, and other disorders. SBMA is caused by an abnormal androgen receptor (AR) protein. Like other PolyA/PolyQ diseases, the abnormal protein is unstable and aggregates within cells, eventually leading to cell death. Patients suffering from SBMA suffer from progressive neuromuscular deterioration that can end up in extreme disability and repeated aspiration pneumonia. There is currently no approved therapy for SBMA.

Studies in cell models have shown that Trehalose in its capacity both as an anti-mutant protein aggregation and as an autophagy enhancer is able to reduce protein aggregates and improve cell survival in SBMA cells. Additional studies show that activation of autophagy may be beneficial in alleviating disease symptoms in animal models.

We have signed an agreement with MINDS (a group within the U.S. National Institutes of Health, or NIH) pursuant to which MINDS will conduct animal studies on SBMA mouse models to validate explore the potential efficacy of Cabaletta . MINDS will conduct the study using its own resources, and we will supply the research material. We have retained all intellectual property and commercialization rights for this indication, and if the animal studies are successful and subject to regulatory approval, we will commence Phase 2/3 clinical trials.

Registered: 01-08-2013
Posts: 61
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I have found other references to the use of Trehalose and have even managed to acquire a pound for testing, but the jury is out on whether it can be effective taken orally. At least one study has dealt with the effects of truncated segments of polyq AR, a result of incomplete autophagy. Cabaletta his been approved for stage two trials for another bulbar form of MD (http://globenewswire.com/news-release/2015/03/12/714647/10124426/en/BioBlast-Receives-IND-Clearance-to-Commence-Clinical-Trial-for-Cabaletta-for-the-Treatment-of-OPMD.html)
Registered: 01-08-2013
Posts: 61
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Cabaletta (trehalose) has been approved for Stage three trials for OPMD. Here is an NIH link about trehalose and SBMA http://www.ncbi.nlm.nih.gov/pubmed/23810450
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Kennedy's Disease Association
PO Box 1105 Coarsegold CA 93614
Tel: 1-855-532-7762
Email: info@kennedysdisease.org