Sapropterin

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).