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Santhera Pharmaceuticals' investigational congenital muscular dystrophy drug receives orphan drug designation
Saturday, August 16 2008 | Comments
What's This?
The Food and Drug Administration granted orphan drug designation to Santhera Pharmaceuticals' investigational compound, SNT-317/omigapil, for the treatment of congenital muscular dystrophy.Congenital muscular dystrophy refers to a group of inherited neuromuscular disorders characterized by progressive loss of muscle tissue, or hypotonia, and affects infants and young children and leads to life-threatening progressive muscle weakness.
The company said data have demonstrated that the drug prevents apoptosis and ameliorates the pathology of laminin-alpha 2-deficient muscular dystrophy in a disease-relevant model. One common subtype of the disease is associated with mutations in the gene encoding laminin-alpha 2, a protein in the extracellular matrix of muscle cells.
Santhera licensed omigapil from Novartis AG for development in this and other neuromuscular diseases in 2007.
The company said preclinical data will be presented in the United Kingdom at the 13th International World Muscle Society Congress this fall.
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