Results 111 to 120 of about 474 (133)
Molecular Genetics and Metabolism, 2019 OBJECTIVE: To provide recommendations for managing hypersensitivity adverse events (HAEs) to an injectable enzyme substitution therapy (pegvaliase, a PEGylated phenylalanine ammonia lyase enzyme) in adult patients with phenylketonuria (PKU).
Oliver Hausmann +2 more
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Pegvaliase dosing in adults with PKU: Requisite dose for efficacy decreases over time
Molecular Genetics and Metabolism, 2022 Novel pharmaceutical therapies such as pegvaliase, phenylalanine ammonia lyase (PAL), have enhanced disease control for many individuals with phenylketonuria (PKU). We present a retrospective chart review to assess pegvaliase doses over time in individuals followed at the Boston Children's Hospital PAL Clinic, including those who started pegvaliase in ...
Suzanne Hollander, Stephanie Sacharow
exaly +3 more sources
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Genetics in Medicine
Given the complexity and close monitoring needs of pegvaliase, we evaluated the real-life clinical outcomes of patients with phenylketonuria (PKU) managed in a pharmacist-led pegvaliase pharmacotherapy program.A review of 51 PKU patients initiated on pegvaliase at the PKU clinic of M Health Fairview, Minneapolis, MN, between May 2018 and May 2024 was ...
Nishitha R Pillai, Alia Ahmed
exaly +3 more sources
Given the complexity and close monitoring needs of pegvaliase, we evaluated the real-life clinical outcomes of patients with phenylketonuria (PKU) managed in a pharmacist-led pegvaliase pharmacotherapy program.A review of 51 PKU patients initiated on pegvaliase at the PKU clinic of M Health Fairview, Minneapolis, MN, between May 2018 and May 2024 was ...
Nishitha R Pillai, Alia Ahmed
exaly +3 more sources
Canadian Journal of Health Technologies, 2022
CADTH recommends that Palynziq should be reimbursed by public drug plans for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 µmol/L) on existing management if certain conditions are met. Palynziq should only be covered ...
openaire +1 more source
CADTH recommends that Palynziq should be reimbursed by public drug plans for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 µmol/L) on existing management if certain conditions are met. Palynziq should only be covered ...
openaire +1 more source
Best practice recommendations for the management of anxiety during the pegvaliase journey
Molecular Genetics and MetabolismPegvaliase, an enzyme substitution therapy, is a treatment option for phenylketonuria (PKU). Due to the neuropathophysiology and disease burden of PKU, individuals can experience baseline anxiety unrelated to pegvaliase therapy. In addition, there are aspects of pegvaliase therapy that may be anxiety-inducing for those considering or receiving ...
Suzanne Hollander +2 more
exaly +3 more sources
Pegvaliase: First Global Approval
BioDrugs, 2018BioMarin Pharmaceutical is developing pegvaliase (PALYNZIQ™) as a treatment for phenylketonuria, a genetic disorder caused by deficiency of phenylalanine hydroxylase which leads to neurotoxic accumulation of phenylalanine. Data from the phase III PRISM clinical trial program indicate treatment with pegvaliase is associated with sustained reductions in ...
openaire +2 more sources
Journal of Immunological Methods, 2019
Pegvaliase is an enzyme substitution therapy developed to lower blood phenylalanine (Phe) in adults with phenylketonuria (PKU). In phase 3 clinical studies, pegvaliase substantially reduced mean blood Phe in adult subjects with PKU. The most common type of adverse event observed in the pegvaliase clinical program was hypersensitivity adverse events ...
Kevin Larimore +8 more
openaire +2 more sources
Pegvaliase is an enzyme substitution therapy developed to lower blood phenylalanine (Phe) in adults with phenylketonuria (PKU). In phase 3 clinical studies, pegvaliase substantially reduced mean blood Phe in adult subjects with PKU. The most common type of adverse event observed in the pegvaliase clinical program was hypersensitivity adverse events ...
Kevin Larimore +8 more
openaire +2 more sources
Pegvaliase therapy in sapropterin-responsive PKU patients
Molecular Genetics and Metabolism, 2022ER Vucko +7 more
openaire +1 more source