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recommendations

Pegvaliase (PALYNZIQ®) is an enzyme injected under theskin to lower blood phenylalanine (Phe) levels in adults withphenylketonuria (PKU). PKU is a condition in which elevatedlevels of Phe in the blood and brain can affect the way a personthinks, feels and acts, and may impact their daily life. It iscommon for adults with PKU to experience anxiety. This can becaused by PKU itself as well as its management.

26th Jun 2024

A plain language summary: Management considerations for people with phenylketonuria and anxiety during the pegvaliase journey

Rett syndrome is a rare genetic disorder that affects the way the brain develops. The medication trofinetide (DAYBUE™) was studied in a large clinical trial called LAVENDER, where it showed a benefit in reducing symptoms of Rett syndrome versus placebo (placebo did not contain medication but looked the same as trofinetide and was taken in the same way). The most common side effect in the trial was diarrhea (frequent and/or watery bowel movements). In order to help caregivers and healthcare providers, experts created recommendations on how to prevent and manage diarrhea if it occurs during trofinetide treatment

31st Jul 2023

recommendations

A plain language summary: Management considerations for people with phenylketonuria and anxiety during the pegvaliase journey

Plain Language Summary: recommendations for managing diarrhea caused by trofinetide use in individuals with Rett Syndrome