ENZYME TREATMENT

Until recently, there was no treatment option for PKU patients in our country, other than sapropterin dihydrochloride, which is diet and drug therapy. Even though phenylalanine levels are brought under control with diet therapy, there are difficulties in compliance with diet; drug therapy does not give results in every individual with PKU. For this reason, studies on alternative methods to diet and drug treatment are continuing rapidly.

Patients with PKU cannot produce an enzyme called "FAH" secreted from the liver due to a defect in the gene. With the “PEG-PAL” enzyme treatment approved by the FDA in May 2018, this enzyme, which is found to be inadequate in patients with PKU, is provided to the body from the outside.