Dr. Matthew Ellinwood, an animal science professor at Iowa State University and Dr. Patricia Dickson, with the Harbor-UCLA Medical Center, have been researching a new study that brings hope to children with the rare genetic disorder called mucopolysaccharidosis type I, or MPS I. MPS I is caused by the lack of an important enzyme that breaks down substances that the body needs in order to build strong and normal nerves, bones, cartilage, etc.
Normally, research done on trying to solve this problem with MPS I is minimal and can only do so much. But, Ellinwood, who has been studying this disease in dogs for 12 years, claims that the study they have been working on “outlines the potential to fully treat mucopolysaccharidosis type I using current technology and medicines, and prevent what might otherwise be the fatal or seriously debilitating consequence of this inherited disease.” Which, is something no one else has ever been able to do before.
Now, there is no cure for severe forms of MPS I or similar disorders, but this research is making new discoveries that can improve methods of enzyme delivery in human patients with similar genetic disorders. And with this new study, babies could also be tested for this disorder at the same time they are tested for other inherited diseases. All in all, this is a groundbreaking discovery that could potentially help out all patients with MPS I.
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