Yale Medicine is now open as a clinical trial site for a study called the “Safety and Tolerability of RTX-134 in Adults with Phenylketonuria,” which is an engineered red blood cell containing the enzyme PAL:  The site is actively recruiting adult participants (18 years or older) to try this investigational medication, which is administered via a short IV infusion.  Please refer all questions about this study to Lori Melancon in Patient Advocacy at Rubius Therapeutics at, visit or visit www.ClinicalTrials.Gov and search for study NCT04110496.



CLINICAL TRIAL at Boston Children’s Hospital - Ages 18-64


Boston Children’s Hospital is now open as a clinical trial site for the study called Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria (PKU,) which has been referred to as the “PKU Probiotic Study.” They are actively recruiting adult participants (age 18-64) to try an investigational medication which is taken by mouth.


Please see the attached flier. Please refer all questions about this study to Kendall Davis at (919) 788-6519 or visit ClinicalTrials.Gov and search for study NCT03516487


Share your story - ALL AGES - Individuals and Families

As PKU Day is approaching the Synlogic team has decided to dedicate one of its meeting rooms as the ‘PKU Room’. Synlogic would love to decorate it with photos of people living with PKU, and their stories. Please see attached example of a small "PKU Star” poster that Synlogic will create for each individual/family to inspire the Synlogic team as they develop novel therapies to help the community. Please send all submissions by Friday, December 21, 2018 to Majar Puurunen at or feel free to respond to this email with it and NECPAD will pass it along. Thank you! 

***NOTE: NECPAD strives to remain neutral when communicating information        relevant to our PKU/AD community. NECPAD wants to ensure our                    community is kept well informed of news related to PKU/AD but in no way        does NECPAD endorse any clinical trial, treatment, product, industry                partner, or event.

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