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- Hurler-Sheie - Genetic Disorder Therapy Likely (Ryan Foundation)
"The idea of using enzyme replacement therapy for genetic diseases such as Hurler/Scheie has been around for a long time, 'but we despaired of ever going to clinical trials because it costs a lot of money,' said Neufeld, who discovered the mutated gene that causes MPS-1."
The parents of a child with the genetic disorder put together a foundation and raised over $1 million for research. An effective therapy seems to have been found as a direct result of their funding efforts. "Children with the disorder - about one in every 25,000 born in the United States - are missing an enzyme called alpha-L-iduronidase that breaks down mucopolysaccharides." 6-02
Research
- Heart Muscle Regenerates (USA Today)
Provides a summary of a major medical finding, that a heart can repair itself after a heart attack. Before this, scientists and doctors believed that heart muscles could not be restored after they were damaged in a heart attack. 6-01
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