New PKU Drug Already Benefiting Adults
The recently approved phenylketonuria (PKU) drug, pegvaliase, is helping patients experience less disease burden—and drastically in some cases.
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Palynziq, Newly Approved for Adults With PKU, Performs Well in Clinical Trial
Palynziq is specifically intended for use by patients with PKU over the age of 18 who have blood Phe levels at or above 600 μmol/L while following their current therapies.
New Therapies on the Horizon for Huntington's Disease
Several promising new treatment developments are offering a ray of hope to Huntington’s patients, their at-risk children, and the clinicians who treat them.