المؤلفون: Stepien, Karolina M.^{Aff1, IDs13023023027961_cor1}, Broomfield, Alexander, Cole, Duncan, Deegan, Patrick B., Forshaw-Hulme, Stuart, Hughes, Derralynn, Jovanovic, Ana, Morris, Liz, Muir, Alison, Ramaswami, Uma

المصدر: Orphanet Journal of Rare Diseases. 18(1)

المؤلفون: Wallace, Eric L., Goker-Alpan, Ozlem, Wilcox, William R., Holida, Myrl, Bernat, John, Longo, Nicola, Linhart, Aleš, Hughes, Derralynn A., Hopkin, Robert J., Tøndel, Camilla, Langeveld, Mirjam, Giraldo, Pilar, Pisani, Antonio, Germain, Dominique Paul, Mehta, Ankit, Deegan, Patrick B., Molnar, Maria Judit, Ortiz, Damara, Jovanovic, Ana, Muriello, Michael

المصدر: Journal of Medical Genetics; Jun2024, Vol. 61 Issue 6, p520-13, 24p

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المؤلفون: Valayannopoulos, Vassili, Malinova, Vera, Honzík, Tomas, Balwani, Manisha, Breen, Catherine, Deegan, Patrick B, Enns, Gregory M, Jones, Simon A, Kane, John P, Stock, Eveline O, Tripuraneni, Radhika, Eckert, Stephen, Schneider, Eugene, Hamilton, Gavin, Middleton, Michael S, Sirlin, Claude, Kessler, Bruce, Bourdon, Christopher, Boyadjiev, Simeon A, Sharma, Reena, Twelves, Chris, Whitley, Chester B, Quinn, Anthony G

المصدر: Journal of hepatology. 61(5)

مصطلحات موضوعية: Liver, Humans, Wolman Disease, Alanine Transaminase, Aspartate Aminotransferases, Lipids, Recombinant Proteins, Drug Administration Schedule, Adult, Middle Aged, Female, Male, Sterol Esterase, Young Adult, Dyslipidemia, Enzyme replacement, Fatty liver, Hepatomegaly, Lysosomal storage, Gastroenterology & Hepatology, Clinical Sciences, Public Health and Health Services

URL الوصول: https://escholarship.org/uc/item/7jr1844c

المؤلفون: Balwani, Manisha, Breen, Catherine, Enns, Gregory M, Deegan, Patrick B, Honzík, Tomas, Jones, Simon, Kane, John P, Malinova, Vera, Sharma, Reena, Stock, Eveline O, Valayannopoulos, Vassili, Wraith, J Edmond, Burg, Jennifer, Eckert, Stephen, Schneider, Eugene, Quinn, Anthony G

المصدر: Hepatology. 58(3)

مصطلحات موضوعية: Rare Diseases, Clinical Research, Clinical Trials and Supportive Activities, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Adult, Alanine Transaminase, Aspartate Aminotransferases, Cholesterol Ester Storage Disease, Cholesterol, HDL, Cholesterol, LDL, Dose-Response Relationship, Drug, Female, Humans, Liver, Male, Middle Aged, Recombinant Proteins, Sterol Esterase, Treatment Outcome, Triglycerides, Medical Biochemistry and Metabolomics, Clinical Sciences, Immunology, Gastroenterology & Hepatology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/4b21j37t

المؤلفون: Mehta, Ankit, Eyskens, Francois J., Pisani, Antonio, Feldt-Rasmussen, Ulla, Hughes, Derralynn, Deegan, Patrick B., Goker-Alpan, Ozlem, Wallace, Eric, Bernat, John, Longo, Nicola, Bianchi, Francesco, Rocco, Rossana, Paleari, Ylenia

المصدر: In Molecular Genetics and Metabolism February 2024 141(2)

المؤلفون: Roberts, Mark E., Cole, Duncan, Deegan, Patrick B., Geberhiwot, Tarekegn, Hughes, Derralynn, Lachmann, Robin, Sharma, Reena, Jain, Vipul, Moffat, Elizabeth, Rutecki, Jasmine, Clarke, Sophie

المصدر: In Molecular Genetics and Metabolism February 2024 141(2)

المؤلفون: Hopkin, Robert J., Ganesh, Jaya, Bernat, John, Goker-Alpan, Ozlem, Nicholls, Kathleen, Pahl, Madeleine V., Deegan, Patrick B., Whitley, Chester B., Hughes, Derralynn, Cao, Liching, Chen, Michael, Shiue, Lisa, Rojkjaer, Lisa, Wilcox, William R.

المصدر: In Molecular Genetics and Metabolism February 2024 141(2)

المؤلفون: Bernat, John, Holida, Myrl D., Longo, Nicola, Goker-alpan, Ozlem, Wallace, Eric, Deegan, Patrick B., Tøndel, Camilla, Eyskens, Francois J., Feldt-rasmussen, Ulla, Hughes, Derralynn, Pisani, Antonio, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G., Waldek, Stephen, Wilcox, William R.

المصدر: Bernat , J , Holida , M D , Longo , N , Goker-alpan , O , Wallace , E , Deegan , P B , Tøndel , C , Eyskens , F J , Feldt-rasmussen , U , Hughes , D , Pisani , A , Rocco , R , Almon , E B , Alon , S , Chertkoff , R , Warnock , D G , Waldek , S & Wilcox , W R 2023 , ' Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease : Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study ' , Molecular Genetics and Metabolism , vol. 138 , no. 2 , 107027 , pp. 16 .

مصطلحات الفهرس: conferenceObject

URL: https://curis.ku.dk/portal/da/publications/longterm-safety-and-efficacy-of-pegunigalsidase-alfa-administered-every-4-weeks-in-patients-with-fabry-disease(e2e12101-a912-4f45-925e-db2db3df8a32).html
https://doi.org/10.1016/j.ymgme.2022.107027

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