GTE therapy is offered to all boys with the biochemical defect of ALD. This therapy reverses the principal biochemical abnormality, namely, the accumulation of very long chain fatty acids. We are encouraged by the fact that all the pts. who were neurologically symptomatic at the beginning of therapy have remained so & that their brain MRI studies also have remained stable. Mean duration of therapy is 14 months. Longer follow-up is required for definitive conclusion.

Project Start
1999-12-01
Project End
2000-11-30
Budget Start
Budget End
Support Year
39
Fiscal Year
2000
Total Cost
Indirect Cost
Name
Johns Hopkins University
Department
Type
DUNS #
045911138
City
Baltimore
State
MD
Country
United States
Zip Code
21218
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