Phenylketonuria awareness and access to treatment (No. 2)
Primary sponsor
Liz Twist
Motion Text
That this House recognises the progress made in improving the care of people with phenylketonuria (PKU), a rare inherited metabolic disorder which prevents the body from properly metabolising phenylalanine; welcomes that many patients have benefitted from access to sapropterin, which has improved quality of life for some individuals living with PKU; notes, however, that significant unmet clinical needs remain, with many patients unable to benefit from existing treatments and still reliant on a highly restrictive diet; further recognises the continuing impact of PKU on patients, carers and families; and highlights the need for access to further innovative treatments and technologies that can improve outcomes and reduce the lifelong burden of PKU.