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1.
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Qual Life Res;
30(10): 2983-2994, 2021 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-33914257
2.
Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Mol Genet Metab;
131(1-2): 219-228, 2020.
Artículo
en Inglés
| MEDLINE | ID: mdl-33012654
3.
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
Clin Exp Nephrol;
24(2): 157-166, 2020 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-31889231
4.
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 randomized, multicenter, double-blind clinical trial and extension study.
Genet Med;
21(9): 1987-1997, 2019 09.
Artículo
en Inglés
| MEDLINE | ID: mdl-30723321
5.
Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment.
J Med Genet;
54(11): 781-786, 2017 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-28756410
6.
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study.
Am J Med Genet A;
179(6): 1069-1073, 2019 06.
Artículo
en Inglés
| MEDLINE | ID: mdl-30920142
7.
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
Hum Gene Ther;
34(13-14): 605-615, 2023 07.
Artículo
en Inglés
| MEDLINE | ID: mdl-37166361
8.
Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study.
Orphanet J Rare Dis;
15(1): 190, 2020 07 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-32693833
9.
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 trial (ESSENCE study).
Orphanet J Rare Dis;
15(1): 158, 2020 06 23.
Artículo
en Inglés
| MEDLINE | ID: mdl-32576219
10.
Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report.
Case Rep Obstet Gynecol;
2019: 1030259, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-31934472
11.
Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
Orphanet J Rare Dis;
13(1): 68, 2018 04 27.
Artículo
en Inglés
| MEDLINE | ID: mdl-29703262
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