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).
Kennedy's Disease is an adult-onset disease with symptoms usually appearing between the ages of 30 and 50.
Given recent evidence that Kennedy's Disease may affect muscles directly, causing them to become weak independent of the nervous system, such modulators have the potential to improve motor function in patients affected by KD (SBMA).
We are very pleased to have been selected by the KDA to receive funding which will support the advancement of our scientific platform against Kennedy's Disease, complementing our lead Rycal programs for heart failure and Duchenne Muscular Dystrophy," said Dr.
Upon granting the award, the Kennedy's Disease Association commented, "The KDA is pleased to be able to partner with ARMGO, as part of our overall effort to cure KD.
In lieu of flowers, please donate to the Kennedy's Disease
Researchers have shown that in a fourth CAG-repeat illness, Kennedy's disease, the expanded gene pumps out flawed versions of its normal protein, a receptor for the hormone androgen.
Groups from France, Japan, and the United States report in the May Nature Genetics that Huntington's disease, DRPLA, and SCA1 follow in the footsteps of Kennedy's disease, not fragile X.
Lloyd endured the effects of Kennedy's Disease
for many years; he died at home.
Through a joint research effort with the Neurogenetics Branch of the NINDS, ASC will use the funding to pursue development of an oral drug treatment for spinal and bulbar muscular atrophy (SBMA) or Kennedy's Disease, a rare hereditary neurodegenerative disease, which currently has no approved drug available to patients.
About Spinal Bulbar Muscular Atrophy (SBMA) - Kennedy's Disease
The study was funded in part by the Muscular Dystrophy Association, the Kennedy's Disease
Association, the National Institutes of Health and ALSAC.