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Matthew Evangelista was 11 years old when he was diagnosed with an extremely rare degenerative disease called mucopolysaccharidosis type VII or sly syndrome.
Many patients suffering from sly syndrome die at birth or soon after while a few live into their early adulthood.
MPS VII, Sly syndrome is a progressive and debilitating inherited, rare genetic disease, which has previously had no approved therapy.
company is formulating various possible treatments for rare or extremely rare diseases, comprising Sly Syndrome, a cellular and organ dysfunction that typically leads to death by early adulthood.
After 3 weeks, the transplanted cells had penetrated diverse regions of the brain and produced beta-glucuronidase, the enzyme that victims of Sly syndrome cannot make.
M2 EQUITYBITES-November 16, 2017-Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome
M2 PHARMA-November 16, 2017-Ultragenyx passes US FDA's approval for Mepsevii for the rare genetic enzyme disorder Sly syndrome