The National Institute for Health and Care Excellence (NICE) has issued draft guidance that recommends Kuvan (sapropterin), alongside a low-phenylalanine diet, ... Read more
A first-of-its-kind pig model of phenylketonuria (PKU) has been developed that could be useful in advancing potential disease treatments, including gene therapies. ... Read more
Homology Medicines, which specializes in gene therapies for rare diseases and is developing two potential such therapies — HMI-102 and HMI-103 — for people with phenylketonuria (PKU), ... Read more
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with ... Read more
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option ... Read more
Genetic sequencing alone is not sufficient for newborn screening of metabolic disorders such as phenylketonuria, a new study indicates. The study, ... Read more
Using extracts from plants in oral capsule formulations could lead to enzyme therapies that resist the acidic environment of the stomach and ... Read more
A planned Phase 2 clinical trial of Synlogic’s SYNB1618, an investigational treatment for people with phenylketonuria (PKU), is expected to be delayed ... Read more
