P371 Switching treatment to cipaglucosidase alfa+miglustat positively affects motor function and quality of life in patients with late-onset Pompe disease
Claeys, K., Byrne, B., Diaz-Manera, J., Dimachkie, M., Kishnani, P., Kushlaf, H., Mozaffar, T., Roberts, M., Schoser, B., Hummel, N., Holdbrook, F., Raza, S., Shohet, S., Wasfi, Y., Toscano, A.
Published in Neuromuscular disorders : NMD (01.10.2023)
Published in Neuromuscular disorders : NMD (01.10.2023)
Get full text
Journal Article
P373 Effect size analysis of cipaglucosidase alfa + miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL
Diaz-Manera, J., Bratkovic, D., Byrne, B., Claeys, K., Dimachkie, M., Kushlaf, H., Kishnani, P., Laforêt, P., Mozaffar, T., Roberts, M., Toscano, A., Castelli, J., Raza, S., Holdbrook, F., Das, S Sitaraman, Wasfi, Y., Schoser, B.
Published in Neuromuscular disorders : NMD (01.10.2023)
Published in Neuromuscular disorders : NMD (01.10.2023)
Get full text
Journal Article
P156 Muscle biopsy findings in a large cohort of patients affected by valosin containing protein disease: preliminary analysis of the international multicentric VCP study
Schiava, M., Nishino, I., Inoue, M., Nishimori, Y., Saito, Y., Polvikoski, T., Charlton, R., Parkhurst, Y., Henderson, M., Marini-Bettolo, C., Guglieri, M., Straub, V., Weihl, C., Stojkovic, T., Villar-Quiles, R., Romero, N., Evangelista, T., Pegoraro, E., De Bleecker, J., Monforte, M., Malfatti, E., Souvannanorath, S., Severa, G., Alonso-Jiménez, A., Baets, J., De Ridder, W., De Jonghe, P., Kierdaszuk, B., Claeys, K., Muelas, N., Oldfors, A., Rodolico, C., Quin, C., Dominguez, C., Lain, A Herández, Pál, E., Papadimas, G., Kushlaf, H., Alfano, L., Alonso-Pérez, J., Luo, S., Badrising, U., Bevilacqua, J., Nedkova-Hristova, V., Cetin, H., Gelpi, E., Klotz, S., Plana, M Olivé
Published in Neuromuscular disorders : NMD (01.10.2023)
Published in Neuromuscular disorders : NMD (01.10.2023)
Get full text
Journal Article
P.025 Efficacy and safety results of the avalglucosidase alfa phase 3 COMET trial in participants with late-onset Pompe disease (LOPD)
Tarnopolsky, M, Attarian, S, Borges, J, Bouhour, F, Choi, Y, Clemens, P, Day, J, Díaz-Manera, J, Erdem-Ozdamar, S, Goker-Alpan, O, Illarioshkin, S, Kishnani, PS, Kostera-Pruszczyk, A, Kushlaf, H, Ladha, S, Mozaffar, T, Roberts, M, Straub, V, Toscano, A, van der Ploeg, AT, An Haack, K, Hug, C, Huynh-Ba, O, Johnson, J, Zhou, T, Dimachkie, MM, Schoser, B
Published in Canadian journal of neurological sciences (01.11.2021)
Published in Canadian journal of neurological sciences (01.11.2021)
Get full text
Journal Article