Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment

por Mauer, Michael, Sokolovskiy, Alexey, Barth, Jay A, Castelli, Jeffrey P, Williams, Hadis N, Benjamin, Elfrida R, Najafian, Behzad
Publicado 2017

Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study

por Narita, Ichiei, Ohashi, Toya, Sakai, Norio, Hamazaki, Takashi, Skuban, Nina, Castelli, Jeffrey P., Lagast, Hjalmar, Barth, Jay A.
Publicado 2019

FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument

por Shields, Alan L., Lamoureux, Roger E., Taylor, Fiona, Barth, Jay A., Mulberg, Andrew E., Kessler, Vivian, Skuban, Nina
Publicado 2021

Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study

por Murrell, Dedee F., Paller, Amy S., Bodemer, Christine, Browning, John, Nikolic, Milos, Barth, Jay A., Lagast, Hjalmar, Krusinska, Eva, Reha, Allen
Publicado 2020

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled...

por Paller, Amy S., Browning, John, Nikolic, Milos, Bodemer, Christine, Murrell, Dedee F., Lenon, Willistine, Krusinska, Eva, Reha, Allen, Lagast, Hjalmar, Barth, Jay A.
Publicado 2020

Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report

por Haninger-Vacariu, Natalja, El-Hadi, Sarah, Pauler, Udo, Foretnik, Marina, Kain, Renate, Prohászka, Zoltán, Schmidt, Alice, Skuban, Nina, Barth, Jay A., Sunder-Plassmann, Gere
Publicado 2019

Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study

por Hughes, Derralynn A., Nicholls, Kathleen, Sunder‐Plassmann, Gere, Jovanovic, Ana, Feldt‐Rasmussen, Ulla, Schiffmann, Raphael, Giugliani, Robert, Jain, Vipul, Viereck, Chris, Castelli, Jeffrey P., Skuban, Nina, Barth, Jay A., Bichet, Daniel G.
Publicado 2019

Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3...

por Germain, Dominique P., Nicholls, Kathy, Giugliani, Roberto, Bichet, Daniel G., Hughes, Derralynn A., Barisoni, Laura M., Colvin, Robert B., Jennette, J. Charles, Skuban, Nina, Castelli, Jeffrey P., Benjamin, Elfrida, Barth, Jay A., Viereck, Christopher
Publicado 2019

Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

por Schiffmann, Raphael, Bichet, Daniel G., Jovanovic, Ana, Hughes, Derralynn A., Giugliani, Roberto, Feldt-Rasmussen, Ulla, Shankar, Suma P., Barisoni, Laura, Colvin, Robert B., Jennette, J. Charles, Holdbrook, Fred, Mulberg, Andrew, Castelli, Jeffrey P., Skuban, Nina, Barth, Jay A., Nicholls, Kathleen
Publicado 2018