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ARMGO Pharma Receives Research Grant Award from Kennedy's Disease Association to Support Advancement of Rycal Compounds
Date:11/6/2013

TARRYTOWN, N.Y. and COARSEGOLD, Calif., Nov. 6, 2013 /PRNewswire/ -- ARMGO Pharma and the Kennedy's Disease Association (KDA) today announced that a research grant has been awarded to support preclinical work on novel, orally available compounds that have potential as a novel treatment for Kennedy's Disease (also known as Spinal Bulbar Muscular Atrophy or SBMA). The project being advanced by ARMGO Pharma is focused on a novel class of small molecule drugs known as Rycals® which target the Ryanodine Receptor (RyR), an intracellular calcium channel that becomes leaky in disease states, including Kennedy's Disease, and contributes to loss of muscle strength and function.

Kennedy's Disease (KD) is a rare and currently incurable and non-treatable recessive X-linked, genetic progressive neuromuscular disease. Spinal and bulbar motor neurons are primarily affected causing muscle weakness and wasting (atrophy) throughout the body including the extremities (legs/arms) as well as in the face and throat. This results in speech and swallowing difficulties as well as major motor deficiencies. Kennedy's Disease is an adult-onset disease with symptoms usually appearing between the ages of 30 and 50.  It is estimated that 1 in 40,000 male individuals worldwide have the disease; however many go undiagnosed or misdiagnosed for years. The most frequent misdiagnosis is the fatal Lou Gehrig's Disease (ALS).

The focus of the research project is to assess the therapeutic effects of orally bioavailable, small molecule modulators of the RyR (known as Rycals) which repair pathophysiologic calcium leak in skeletal muscle disease to reverse chronic muscle weakness. Given recent evidence that Kennedy's Disease may
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SOURCE ARMGO Pharma; Kennedy's Disease Association
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