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Denmark LNAA STUDY 1 tablet/kg/day

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Denmark LNAA STUDY 1 tablet/kg/day

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  1. UkraineProf. Elena Grechanina, Kharkov Genetic & Neonatal Screening Centre RussiaProf. Peter Novikov, Moscow Central Genetic Center DenmarkFlemming Güttler, M.D., PhD., Glostrup, J. F. Kennedy Genetic InstituteUSAReuben Matalon, M.D., Ph.D., Kim Matalon, the University of Texas Medical Branch

  2. Denmark LNAA STUDY1 tablet/kg/day

  3. Denmark LNAA STUDY2 tablets/kg/day

  4. Russia LNAA STUDY

  5. Russia LNAA STUDY

  6. Russia LNAA Study

  7. USA LNAA STUDY

  8. USA LNAA STUDY

  9. µmol/l1260 870 960 474 762 426 780

  10. µmol/l1200 828 765 624

  11. US Blood Phe and TyrNeoPhe Patient K 1 Weekµmol/L (mg)

  12. US Blood Phe and TyrNeoPhe Patient G 1 Weekµmol/L (mg)

  13. NeoPhe 0.5 g/kg in PKU Subjects • 13 subjects • Mean age 26.6 years • 7 males, 6 females • Mean decrease in blood Phe after one week 243 µmol/L • Average decrease in blood Phe 22 %.

  14. NeoPhe 1.0 g/kg in PKU Subjects • 7 subjects • Mean age 25.2 years • 5 males, 2 females • Mean decrease in blood Phe after one week 377 µmol/L • Average decrease in blood Phe 25 %.

  15. Figure 1. Blood Phe Response to 0.5g/kg NeoPhe in Patients withPKU Paired t-test: p=0.001

  16. Figure 2. Blood Phe Response to 1.0 g/kg NeoPhe in Patients withPKU Paired t-test: p=0.006

  17. CONCLUSIONS: • For the first time mixture of LNAA can lower blood phenylalanine • Using NeoPhe avoids lysine deficiency and negative nitrogen balance • NeoPhe offers new options to treat PKU • Diet can be more liberal with NeoPhe • Still blood phe- levels need to be controlled

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