Sapropterin: useful documents

The patient information sheets are still being worked on, and comments are being gathered.The final version will be available once finished early January. Please check back here often (04 Jan 2021).

Please send your comments to Rachel Skeath - see BIMDG Committee on the contacts page (About > Contacts).

NHS England announced in December 2021 that it will make sapropterin available to individuals with PKU of all ages. In order to facilitate a smooth roll-out of responsiveness testing and prescribing a working group within BIMDG was formed, under the leadership of Rachel Skeath, to develop an agreed pathway, along with patient and parent/guardian information sheets.

Many thanks to the working group - a true multidisciplinary effort - for putting these documents together under considerable time pressure (30 December 2021).

Document Downloads

Consensus pathway for commencing Sapropterin

Sapropterin information sheet for adolescents and adults with PKU

Sapropterin information sheet for Parents and Guardians