Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study

Francjan van Spronsen, Heidi Peters, Lali Margvelashvili, Dodo Agladze, Ida Vanessa D. Schwartz, Maria Giżewska, Takashi Hamazaki, Laura Guilder, Anita MacDonald, Suresh Vijay, Anita Inwood, Maria Minami, Olivia Fjellbirkeland, Allan Lund, Melissa Lah, Janet A. Thomas, Nicola Longo, Ertuğrul Kiykim, Mika Ishige, Alberto Burlina, Amaya Bélanger-Quintana, Frank Rutsch, Thomas Opladen, Halise Mungan, Fatih Ezgü, Drago Bratkovic, Anupam Chakrapani, Tetsuya Ito, Arlindo Guimas, Roberto Zori, Michel Tchan, Stephanie Sacharow, Anabela Oliveira, Patricia Janeiro, Ixiu-Cabrales Guerra, Jaume Campistol Plana, Yılmaz Yıldız, Ebru Canda, Aneal Khan, Jerry Vockley, Margo Sheck Breilyn, Filippo Manti, Alex Larkin, Catalina Hughes, Emelline Liu, Lan Gao, Kimberly Ingalls, Neil Smith, Ania C. Muntau

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DOI: 10.1016/j.gim.2026.101683
Published: 2026-04
Container: Genetics in Medicine
Publisher: Elsevier BV
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/j.gim.2026.101683,
  title = {Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study},
  author = {Francjan van Spronsen and Heidi Peters and Lali Margvelashvili and Dodo Agladze and Ida Vanessa D. Schwartz and Maria Giżewska and Takashi Hamazaki and Laura Guilder and Anita MacDonald and Suresh Vijay and Anita Inwood and Maria Minami and Olivia Fjellbirkeland and Allan Lund and Melissa Lah and Janet A. Thomas and Nicola Longo and Ertuğrul Kiykim and Mika Ishige and Alberto Burlina and Amaya Bélanger-Quintana and Frank Rutsch and Thomas Opladen and Halise Mungan and Fatih Ezgü and Drago Bratkovic and Anupam Chakrapani and Tetsuya Ito and Arlindo Guimas and Roberto Zori and Michel Tchan and Stephanie Sacharow and Anabela Oliveira and Patricia Janeiro and Ixiu-Cabrales Guerra and Jaume Campistol Plana and Yılmaz Yıldız and Ebru Canda and Aneal Khan and Jerry Vockley and Margo Sheck Breilyn and Filippo Manti and Alex Larkin and Catalina Hughes and Emelline Liu and Lan Gao and Kimberly Ingalls and Neil Smith and Ania C. Muntau},
  year = {2026},
  journal = {Genetics in Medicine},
  doi = {10.1016/j.gim.2026.101683},
  url = {https://doi.org/10.1016/j.gim.2026.101683}
}

RIS

TY  - JOUR
TI  - Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study
AU  - Francjan van Spronsen
AU  - Heidi Peters
AU  - Lali Margvelashvili
AU  - Dodo Agladze
AU  - Ida Vanessa D. Schwartz
AU  - Maria Giżewska
AU  - Takashi Hamazaki
AU  - Laura Guilder
AU  - Anita MacDonald
AU  - Suresh Vijay
AU  - Anita Inwood
AU  - Maria Minami
AU  - Olivia Fjellbirkeland
AU  - Allan Lund
AU  - Melissa Lah
AU  - Janet A. Thomas
AU  - Nicola Longo
AU  - Ertuğrul Kiykim
AU  - Mika Ishige
AU  - Alberto Burlina
AU  - Amaya Bélanger-Quintana
AU  - Frank Rutsch
AU  - Thomas Opladen
AU  - Halise Mungan
AU  - Fatih Ezgü
AU  - Drago Bratkovic
AU  - Anupam Chakrapani
AU  - Tetsuya Ito
AU  - Arlindo Guimas
AU  - Roberto Zori
AU  - Michel Tchan
AU  - Stephanie Sacharow
AU  - Anabela Oliveira
AU  - Patricia Janeiro
AU  - Ixiu-Cabrales Guerra
AU  - Jaume Campistol Plana
AU  - Yılmaz Yıldız
AU  - Ebru Canda
AU  - Aneal Khan
AU  - Jerry Vockley
AU  - Margo Sheck Breilyn
AU  - Filippo Manti
AU  - Alex Larkin
AU  - Catalina Hughes
AU  - Emelline Liu
AU  - Lan Gao
AU  - Kimberly Ingalls
AU  - Neil Smith
AU  - Ania C. Muntau
PY  - 2026
JO  - Genetics in Medicine
DO  - 10.1016/j.gim.2026.101683
UR  - https://doi.org/10.1016/j.gim.2026.101683
ER  -

APA

Spronsen, F. V., Peters, H., Margvelashvili, L., Agladze, D., Schwartz, I. V. D., Giżewska, M., Hamazaki, T., Guilder, L., MacDonald, A., Vijay, S., Inwood, A., Minami, M., Fjellbirkeland, O., Lund, A., Lah, M., Thomas, J. A., Longo, N., Kiykim, E., Ishige, M., Burlina, A., Bélanger-Quintana, A., Rutsch, F., Opladen, T., Mungan, H., Ezgü, F., Bratkovic, D., Chakrapani, A., Ito, T., Guimas, A., Zori, R., Tchan, M., Sacharow, S., Oliveira, A., Janeiro, P., Guerra, I., Plana, J. C., Yıldız, Y., Canda, E., Khan, A., Vockley, J., Breilyn, M. S., Manti, F., Larkin, A., Hughes, C., Liu, E., Gao, L., Ingalls, K., Smith, N., & Muntau, A. C. (2026). Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study. Genetics in Medicine. https://doi.org/10.1016/j.gim.2026.101683

Source records

crossref · retrieved 2026-09-25T20:05:41.661Z