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We conclude that the study formula is safe and effective for the treatment of classical MSUD.
Red cell membrane omega-3 polyunsaturated fatty acids and serum zinc and selenium levels increased on study formula.
Treatment with study formula increased plasma concentrations of LAT1 substrates and normalized their calculated uptakes into the nervous system.
In 2006 we designed a lysine-free study formula enriched with arginine to selectively block lysine transport across cerebral endothelia and thereby limit glutaryl-CoA production by brain.
Fifteen Old Order Mennonite children were started on study formula between birth and 34 months of age and seen at least monthly in the office.
Between 2006 and present, we treated twelve consecutive children with study formula (LYSx group) while holding all other treatment practices constant.
Patients receiving the study formula had an improvement in their mortality rate, oxygenation, ventilator-free days, ICU-free days, and less development of new organ dysfunction [45].
To determine the 1-year effectiveness and safety of nutritional supplementation with the study formula on linear growth and weight gain in short and lean prepubertal children and to validate the previously reported findings in those initially treated with placebo.
Of those enrolled, 80% were exposed to the study formula.
Dedicated study nutritionists handled study formula preparations and coding.
No adverse events related to the study formula were reported.
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