Results 1 to 10 of about 358 (89)

Reinstitution of pegvaliase therapy during lactation [PDF]

Molecular Genetics and Metabolism Reports, 2022
Pegvaliase, an injectable form of phenylalanine ammonia lyase, is an enzyme substitution therapy for adults with phenylketonuria (PKU). Experience with pegvaliase during lactation is scarce.
Frances Rohr, Ann Wessel, Cary O. Harding, Susan E. Waisbren, Krista Viau, Amy Kritzer   +5 more
doaj   +4 more sources

Lessons learned from 5 years of pegvaliase in US clinics: A case series [PDF]

Molecular Genetics and Metabolism Reports
Objective: To provide insights and strategies for pegvaliase management in challenging cases with phenylketonuria (PKU) based on the first 5 years of experience with pegvaliase in real-world clinical practice.
Erin Cooney, Zineb Ammous, Tricia Bender, Gillian E. Clague, Marilyn Clifford, Angela Crutcher, Laura Davis-Keppen, Kirsten Havens, Melissa Lah, Stephanie Sacharow, Amarilis Sanchez-Valle, Erika Vucko, Bridget Wardley, Leah Wessenberg, Hans C. Andersson   +14 more
doaj   +4 more sources

Pegvaliase dose escalation to 80 mg daily may lead to efficacy in patients who do not exhibit an optimal response at lower doses [PDF]

Molecular Genetics and Metabolism Reports, 2022
In 2018, pegvaliase was approved as the first enzyme substitution treatment for phenylketonuria (PKU) and is now the second medication available for PKU patients since the approval of sapropterin dihydrochloride in 2007.
Erika R. Vucko, Kirsten E. Havens, Joshua J. Baker, Barbara K. Burton   +3 more
doaj   +4 more sources

Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data [PDF]

Molecular Genetics and Metabolism Reports, 2022
Background: Phenylketonuria (PKU) is an inborn metabolic error characterized by a deficiency of the enzyme required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods.
Melissa Lah, Keziah Cook, Dumingu Aparna Gomes, Stephanie Liu, Nadia Tabatabaeepour, Noam Kirson, Er Chen, Kristin Lindstrom, Kaleigh Bulloch Whitehall, Joost Van Backle, Barbara K. Burton   +10 more
doaj   +4 more sources

Long‐Term Safety and Efficacy of Pegvaliase in Japanese Adults With Phenylketonuria: Final Results of a Phase III Trial [PDF]

JIMD Reports
Phenylketonuria (PKU) is an inborn error of metabolism leading to phenylalanine (Phe) accumulation and consequent neurological, neurocognitive, and psychiatric symptoms. Pegvaliase, a pegylated recombinant phenylalanine ammonia lyase that metabolizes Phe,
Yoko Nakajima, Mika Ishige, Tetsuya Ito, Takashi Hamazaki, Mitsuhiro Kuwahara, Lawrence Lee, Haruo Shintaku   +6 more
doaj   +2 more sources

Development of a practical dietitian road map for the nutritional management of phenylketonuria (PKU) patients on pegvaliase

Molecular Genetics and Metabolism Reports, 2021
Background: The metabolic dietitian/nutritionist (hereafter ‘dietitian’) plays an essential role in the nutritional management of patients with phenylketonuria (PKU), including those on pegvaliase.
Júlio César Rocha, Heather Bausell, Amaya Bélanger-Quintana, Laurie Bernstein, Hülya Gökmen-Özel, Alexandra Jung, Anita MacDonald, Fran Rohr, Esther van Dam, Margret Heddrich-Ellerbrok   +9 more
doaj   +3 more sources

Nutrition management of PKU with pegvaliase therapy: update of the web-based PKU nutrition management guideline recommendations [PDF]

Orphanet Journal of Rare Diseases, 2023
Background The web-based GMDI/SERN PKU Nutrition Management Guideline, published before approval of pegvaliase pharmacotherapy, offers guidance for nutrition management of individuals with phenylketonuria (PKU) treated with dietary therapy and/or ...
Amy Cunningham, Fran Rohr, Patricia Splett, Shideh Mofidi, Heather Bausell, Adrya Stembridge, Aileen Kenneson, Rani H. Singh   +7 more
doaj   +2 more sources

Toxicity spectrum of pegvaliase: A pharmacovigilance analysis using the FAERS database [PDF]

Orphanet Journal of Rare Diseases
Objective This research aimed to assess the safety of pegvaliase through analyzed the data from the FAERS database, thus providing a theoretical foundation for the rational and safe application of pegvaliase in clinical settings.
Luyao Xu, Kaili Mao, Songyang Zhong, Huayu Sun, Hongliang Zheng, Zhenling Fu   +5 more
doaj   +2 more sources

Clinical experience with Pegvaliase in phenylketonuria: A retrospective chart review of outcomes, safety, and dosing patterns [PDF]

Molecular Genetics and Metabolism Reports
Background: Phenylketonuria (PKU) is a metabolic disorder caused by phenylalanine hydroxylase (PAH) deficiency, resulting in elevated plasma phenylalanine (Phe) levels.
Jariya Upadia, Grace Noh, Kea Crivelly, Amy Cunningham, Elise Aziz, Hans C. Andersson   +5 more
doaj   +2 more sources

Use of pegvaliase in the management of phenylketonuria: Case series of early experience in US clinics

Molecular Genetics and Metabolism Reports, 2021
Objective: To present a case series that illustrates real-world use of pegvaliase based on the initial experiences of US healthcare providers. Methods: Sixteen healthcare providers from 14 centers across the US with substantial clinical experience in ...
Darius Adams, Hans C. Andersson, Heather Bausell, Kea Crivelly, Caroline Eggerding, Melissa Lah, Joshua Lilienstein, Kristin Lindstrom, Markey McNutt, Joseph W. Ray, Heather Saavedra, Stephanie Sacharow, Danielle Starin, Jennifer Tiffany-Amaro, Janet Thomas, Erika Vucko, Leah B. Wessenberg, Kaleigh Whitehall   +17 more
doaj   +3 more sources