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Research Fund 2002

Research Fund 2002

 Dr. Holmes Morton, The Clinic for Special Children in Strasburg, PA

In 2002, the MACPAD Board of Directors granted $24,200 to Dr. Holmes Morton of The Clinic for Special Children in Strasburg, PA. Dr. Morton proposes to do a 12 month study on the use of tetrahydrobiopterin (BH4) to determine the effect of biopterin on amino acid tolerence and neurological signs in his PKU patients. He will also determine the mutation of the PAH gene that each of his patients has and which mutations respond to the BH4 doses. He will also study the effects of BH4 supplementation over a 12 month period upon the behavioral and psychiatric problems of PKU patients over 15 yrs. of age. This will include some retarded adult patients with PKU who are currently untreated.

Dr. Barbara Burton, Children's Memorial Hospital in Chicago, IL.

In 2002, MACPAD granted $2,500 to Dr. Barbara Burton of Childrens Memorial Hospital in Chicago, IL. Dr. Burton and staff will work with patients enrolled in her clinic, who are at least 3 years of age. A BH4 loading test will be completed and, in patients found to be responsive, they will be offered continuing treatment of BH4. Plasma phenylalanine levels will be monitored and gradual dietary relaxation will be attempted. Patients will be monitored and dosages of BH4 adjusted as needed. Dr. Burton has participated in several areas of research and has collaborated with Dr. Richard Koch in a study of adults with PKU. MACPAD provided $ Dr. Burton on the last round of funding.