المؤلفون: Kishnani PS, Byrne BJ, Claeys KG, Diaz-Manera J, Dimachkie MM, Kushlaf H, Mozaffar T, Roberts M, Schoser B, Hummel N, Kopiec A, Holdbrook F, Shohet S, Toscano A, Chien Y-H, Loscher W, Laszlo V, Sawada T, Ruck T, Burrow T, Hiwot T, Dearmey S, Wenninger S, Attarian S, Fecarotta S, Sacconi S, Hopkin R, Henderson R, Roxburgh R, Clemens PR, Deegan P, Goker-Alpan O, Musumeci O, Vidal-Fernandez N, Longo N, Freimer M, Tchan M, Tarnopolsky M, Wencel M, Molnar MJ, Gutschmidt K, Kornblum C, Berthy J, Janszky J, Alonso-Perez J, Cauci J, Shin J-H, Avelar J, Vengoechea J, Tarnev I, Kobayashi H, Shiraishi H, Amartino H, Andersen H, Pedro H, Lau H, Akihiro H, Bartosik-Psujek H, Parenti G, Papadimas GK, Kim G, Bouhour F, Butler E, Salort-Campana E, Stefanescu E, Hughes D, Reyes-Leiva D, Bodkin C, Eldridge C, Quinn C, Lindberg C, Tard C, Koritnik B, Khan A, Dominovic-Kovacevic A, Pestronk A, Sebok A

المصدر: Journal of Patient-Reported Outcomes, December 2024

وصف الملف: application/pdf

Relation: https://eprints.ncl.ac.uk/302625; https://eprints.ncl.ac.uk/fulltext.aspx?url=302625/CDBCAC4C-D28B-4C92-AFF8-F4C4E945249B.pdf&pub_id=302625

الاتاحة: https://eprints.ncl.ac.uk/302625

المؤلفون: Rao, V., Byrne, B., Shieh, P., Salabarria, S., Berthy, J., Corti, M., Redican, S., Lawrence, J., Brown, K., Shanks, C., Spector, S., Gonzalez, P., Schneider, J., Morris, C., Clary, C.

المصدر: Neuromuscular Disorders ; volume 31, page S47 ; ISSN 0960-8966

الاتاحة: http://dx.doi.org/10.1016/j.nmd.2021.07.019
https://api.elsevier.com/content/article/PII:S0960896621002066?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0960896621002066?httpAccept=text/plain

المؤلفون: Schoser B., Roberts M., Byrne B. J., Sitaraman S., Jiang H., Laforet P., Toscano A., Castelli J., Diaz-Manera J., Goldman M., van der Ploeg A. T., Bratkovic D., Kuchipudi S., Mozaffar T., Kishnani P. S., Sebok A., Pestronk A., Dominovic-Kovacevic A., Khan A., Koritnik B., Tard C., Lindberg C., Quinn C., Eldridge C., Bodkin C., Reyes-Leiva D., Hughes D., Stefanescu E., SALORT-CAMPANA E., Butler E., Bouhour F., Kim G., Konstantinos Papadimas G., Parenti G., Bartosik-Psujek H., Kushlaf H., Akihiro H., Lau H., Pedro H., Andersen H., Amartino H., Shiraishi H., Kobayashi H., Tarnev I., Vengoechea J., Avelar J., Shin J. -H., Cauci J., Alonso-Perez J., Janszky J., Berthy J., Cornelia K., Gutschmidt K., Claeys K., Judit Molnar M., Wencel M., Tarnopolsky M., Dimachkie M., Tchan M., Freimer M., Longo N., Vidal-Fernandez N., Musumeci O., Goker-Alpan O., Deegan P., Clemens P. R., Roxburgh R., Henderson R., Hopkin R., Sacconi S., Fecarotta S., Attarian S., Wenninger S., Dearmey S., Hiwot T., Burrow T., Ruck T., Sawada T., Laszlo V., Loscher W., Chien Y. -H.

المساهمون: Schoser, B., Roberts, M., Byrne, B. J., Sitaraman, S., Jiang, H., Laforet, P., Toscano, A., Castelli, J., Diaz-Manera, J., Goldman, M., van der Ploeg, A. T., Bratkovic, D., Kuchipudi, S., Mozaffar, T., Kishnani, P. S., Sebok, A., Pestronk, A., Dominovic-Kovacevic, A., Khan, A., Koritnik, B., Tard, C., Lindberg, C., Quinn, C., Eldridge, C., Bodkin, C., Reyes-Leiva, D., Hughes, D., Stefanescu, E., SALORT-CAMPANA, E., Butler, E., Bouhour, F., Kim, G., Konstantinos Papadimas, G., Parenti, G., Bartosik-Psujek, H., Kushlaf, H., Akihiro, H., Lau, H., Pedro, H., Andersen, H., Amartino, H., Shiraishi, H., Kobayashi, H., Tarnev, I., Vengoechea, J., Avelar, J., Shin, J. -H., Cauci, J., Alonso-Perez, J., Janszky, J., Berthy, J., Cornelia, K., Gutschmidt, K., Claeys, K., Judit Molnar, M., Wencel, M., Tarnopolsky, M., Dimachkie, M., Tchan, M., Freimer, M., Longo, N., Vidal-Fernandez, N., Musumeci, O., Goker-Alpan, O., Deegan, P., Clemens, P. R., Roxburgh, R., Henderson, R., Hopkin, R., Sacconi, S., Fecarotta, S., Attarian, S., Wenninger, S., Dearmey, S., Hiwot, T., Burrow, T., Ruck, T., Sawada, T., Laszlo, V., Loscher, W., Chien, Y. -H.

Relation: info:eu-repo/semantics/altIdentifier/wos/WOS:000755602800027; volume:20; issue:12; firstpage:1027; lastpage:1037; numberofpages:11; journal:LANCET NEUROLOGY; http://hdl.handle.net/11588/870280; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85119194577

الاتاحة: http://hdl.handle.net/11588/870280
https://doi.org/10.1016/S1474-4422(21)00331-8

المؤلفون: Marsaudon, E., Berthy, J., Mamoune, S., Deniel, A., Ksiyer, S., Tiuca, D.

المصدر: La Revue de Médecine Interne ; volume 39, issue 3, page 195-199 ; ISSN 0248-8663

الاتاحة: http://dx.doi.org/10.1016/j.revmed.2017.12.002
https://api.elsevier.com/content/article/PII:S0248866317312092?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0248866317312092?httpAccept=text/plain

المؤلفون: Marsaudon, E., Berthy, J., Gautreault, A., Mamoune, S., Boucekkine, R.

المصدر: La Revue de Médecine Interne ; volume 39, issue 10, page 827-828 ; ISSN 0248-8663

الاتاحة: http://dx.doi.org/10.1016/j.revmed.2017.04.001
https://api.elsevier.com/content/article/PII:S0248866317304423?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0248866317304423?httpAccept=text/plain

المؤلفون: Marsaudon, E., Berthy, J., Gautreault, A., Boucekkine, R.

المصدر: La Revue de Médecine Interne ; volume 38, page A144-A145 ; ISSN 0248-8663

الاتاحة: http://dx.doi.org/10.1016/j.revmed.2017.03.189
https://api.elsevier.com/content/article/PII:S0248866317302825?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S0248866317302825?httpAccept=text/plain

المؤلفون: Spencer CT, Bryant RM, Day J, Gonzalez IL, Colan SD, Thompson WR, Berthy J, Redfearn SP, Byrne BJ

المصدر: Pediatrics; Aug2006, Vol. 118 Issue 2, pe337-46, 1p

المؤلفون: Sheela Sitaraman, Richard Roxburgh, Kristina Gutschmidt, Ela Stefanescu, Drago Bratkovic, Thomas Burrow, Kornblum Cornelia, Kristl Claeys, Miriam Freimer, Ozlem Goker-Alpan, Srilakshmi Kuchipudi, Alan Pestronk, Wolfgang Löscher, Francoise Bouhour, Maria Judit Molnar, Ans T. van der Ploeg, Halina Bartosik-Psujek, Mitchell Goldman, Robert D. Henderson, Stephanie Dearmey, Colin Quinn, Paula R. Clemens, Priya S. Kishnani, Jennifer B Avelar, Nicola Longo, Shahram Attarian, Robert Hopkin, Tomo Sawada, Blaž Koritnik, George Konstantinos Papadimas, Hideaki Shiraishi, Christopher Lindberg, Jin-Hong Shin, Ivaylo Tarnev, Tahseen Mozaffar, Heather Lau, Michel Tchan, Jozsef Janszky, Tobias Ruck, Sabrina Sacconi, Benedikt Schoser, Hashiguchi Akihiro, Patrick Deegan, Ernest Butler, Nuria Vidal-Fernandez, Antonio Toscano, Tarekegn Hiwot, Gee Kim, Emmanuelle Salort-Campana, Jeff Castelli, Pascal Laforet, Céline Tard, Crystal Eldridge, Aneal Khan, Stephan Wenninger, Simona Fecarotta, Jordi Díaz-Manera, Jorge Alonso-Pérez, Yin-Hsiu Chien, Mark Tarnopolsky, Olimpia Musumeci, Hiroshi Kobayashi, Helio Pedro, Jonathan Cauci, Agnes Sebok, Cynthia Bodkin, Hai Jiang, Julie Berthy, Vescei Laszlo, Derralynn Hughes, David Reyes-Leiva, Aleksandra Dominovic-Kovacevic, Mazen M. Dimachkie, Hernan Amartino, Hani Kushlaf, Barry J. Byrne, Giancarlo Parenti, Henning Andersen, Mark Roberts, Marie Wencel, Jaime Vengoechea

المساهمون: Schoser, B., Roberts, M., Byrne, B. J., Sitaraman, S., Jiang, H., Laforet, P., Toscano, A., Castelli, J., Diaz-Manera, J., Goldman, M., van der Ploeg, A. T., Bratkovic, D., Kuchipudi, S., Mozaffar, T., Kishnani, P. S., Sebok, A., Pestronk, A., Dominovic-Kovacevic, A., Khan, A., Koritnik, B., Tard, C., Lindberg, C., Quinn, C., Eldridge, C., Bodkin, C., Reyes-Leiva, D., Hughes, D., Stefanescu, E., SALORT-CAMPANA, E., Butler, E., Bouhour, F., Kim, G., Konstantinos Papadimas, G., Parenti, G., Bartosik-Psujek, H., Kushlaf, H., Akihiro, H., Lau, H., Pedro, H., Andersen, H., Amartino, H., Shiraishi, H., Kobayashi, H., Tarnev, I., Vengoechea, J., Avelar, J., Shin, J. -H., Cauci, J., Alonso-Perez, J., Janszky, J., Berthy, J., Cornelia, K., Gutschmidt, K., Claeys, K., Judit Molnar, M., Wencel, M., Tarnopolsky, M., Dimachkie, M., Tchan, M., Freimer, M., Longo, N., Vidal-Fernandez, N., Musumeci, O., Goker-Alpan, O., Deegan, P., Clemens, P. R., Roxburgh, R., Henderson, R., Hopkin, R., Sacconi, S., Fecarotta, S., Attarian, S., Wenninger, S., Dearmey, S., Hiwot, T., Burrow, T., Ruck, T., Sawada, T., Laszlo, V., Loscher, W., Chien, Y. -H., Pediatrics

المصدر: LANCET NEUROLOGY
r-IIB SANT PAU. Repositorio Institucional de Producción Científica del Instituto de Investigación Biomédica Sant Pau
instname
The Lancet Neurology, 20(12), 1027-1037. Lancet Publishing Group
PROPEL Study Group 2021, ' Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL) : an international, randomised, double-blind, parallel-group, phase 3 trial ', The Lancet Neurology, vol. 20, no. 12, pp. 1027-1037 . https://doi.org/10.1016/S1474-4422(21)00331-8

مصطلحات موضوعية: education.field_of_study, medicine.medical_specialty, 1-Deoxynojirimycin, Adolescent, Glycogen Storage Disease Type II, business.industry, Population, alpha-Glucosidases, Enzyme replacement therapy, Placebo, Treatment Outcome, Double-Blind Method, SDG 3 - Good Health and Well-being, Internal medicine, Miglustat, medicine, Clinical endpoint, Humans, Respiratory function, Neurology (clinical), Adverse effect, education, business, Alglucosidase alfa, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7ca8d17d8a65ecd5fd078c358714459b
https://doi.org/10.1016/s1474-4422(21)00331-8