The PAL Clinic is a comprehensive program that cares for patients with phenylketonuria (PKU) who want to receive enzyme therapy. It is part of the Dr. Harvey Levy Program for Phenylketonuria and Related Conditions.
Pegvaliase (Palynziq) is currently the only approved enzyme therapy for PKU. Also known as phenylalanine ammonia lyase (PAL), it is an enzyme substitute for the enzyme that is missing in people with PKU. Pegvaliase has been effective in reducing blood phenylalanine (phe) levels while allowing many individuals with PKU to eat a regular diet.
Our clinic provides thorough education about pegvaliase and offers support and close monitoring throughout your treatment. We will help you become confident with giving yourself pegvaliase injections, will support you through the side effects that can occur, and will help guide you toward your nutrition goals as you undergo pegvaliase treatment. We are your partners in helping you achieve the best possible results.
Your visit to the PAL Clinic
Patients followed in the PAL Clinic are required to have had an appointment with the Boston Children’s Hospital PKU Program within 12 months prior to starting pegvaliase therapy. If it has been more than a year since your last PKU appointment, or if you are new to our center, you can schedule a baseline appointment in the PAL Clinic with Dr. Stephanie Sacharow, Dr. Amy Kritzer, or nurse practitioner Brittany Murray.
Patients interested in initiating pegvaliase treatment will begin with an informational session with a PAL Clinic provider. This discussion includes managing expectations and potential side effects of the drug and completing an online training known as Risk Evaluation and Mitigation Strategies (REMS). This training provides education on the side effects of the medication, including the risk of anaphylaxis.
Initially, we suggest weekly clinic visits for the first few weeks for ongoing injection training and education. Once on treatment, you will be followed closely by a metabolic dietitian experienced in pegvaliase therapy.
Diet monitoring and blood phenylalanine (phe) testing
While starting pegvaliase treatment, it is important to keep your diet stable, continuing the same dietary restrictions, supplements, and medical foods if applicable. We will monitor your blood phe concentrations at least monthly; if they decrease while you are on pegvaliase, then we will guide you in adjusting your diet.
Clinical psychology
Neuropsychological assessments are an important component of care in the PAL Clinic due to PKU’s effect on mood, anxiety, attention, and executive functioning. Visits with our clinical psychologist will help track the cognitive and emotional impact of PKU while you are on pegvaliase therapy.
We recommended having your first assessment when you start the medication, and a repeat assessment after 12 months of treatment. We can schedule additional visits as needed.
Please note that these assessments are optional and are not meant to replace ongoing or long-term mental health services. However, we can provide limited talk therapy and video-talk therapy services upon request.
