Cargando…

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by GLA mutations, resulting in α-galactosidase (α-Gal) deficiency and accumulation of lysosomal substrates. Migalastat, an oral pharmacological chaperone being developed as an alternative to intravenous enzyme replacement the...

Descripción completa

Detalles Bibliográficos
Autores principales:	Hughes, Derralynn A, Nicholls, Kathleen, Shankar, Suma P, Sunder-Plassmann, Gere, Koeller, David, Nedd, Khan, Vockley, Gerard, Hamazaki, Takashi, Lachmann, Robin, Ohashi, Toya, Olivotto, Iacopo, Sakai, Norio, Deegan, Patrick, Dimmock, David, Eyskens, François, Germain, Dominique P, Goker-Alpan, Ozlem, Hachulla, Eric, Jovanovic, Ana, Lourenco, Charles M, Narita, Ichiei, Thomas, Mark, Wilcox, William R, Bichet, Daniel G, Schiffmann, Raphael, Ludington, Elizabeth, Viereck, Christopher, Kirk, John, Yu, Julie, Johnson, Franklin, Boudes, Pol, Benjamin, Elfrida R, Lockhart, David J, Barlow, Carrolee, Skuban, Nina, Castelli, Jeffrey P, Barth, Jay, Feldt-Rasmussen, Ulla
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2017
Materias:	Therapeutics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5502308/ https://www.ncbi.nlm.nih.gov/pubmed/27834756 http://dx.doi.org/10.1136/jmedgenet-2016-104178

Ejemplares similares

Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study
por: Hughes, Derralynn A., et al.
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 randomized, multicenter, double-blind clinical trial and extension study
por: Germain, Dominique P., et al.
Publicado: (2019)

The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
por: Benjamin, Elfrida R., et al.
Publicado: (2017)

Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
por: Bichet, Daniel G., et al.
Publicado: (2021)

The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease
por: Schiffmann, Raphael, et al.
Publicado: (2019)

Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
por: Hughes, Derralynn A, et al.
Publicado: (2023)

Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study
por: Narita, Ichiei, et al.
Publicado: (2019)

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase
por: Warnock, David G., et al.
Publicado: (2015)

Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report
por: Haninger-Vacariu, Natalja, et al.
Publicado: (2019)

Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial
por: Schiffmann, Raphael, et al.
Publicado: (2018)

Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease
por: Bichet, Daniel G., et al.
Publicado: (2020)

Correction: Assessment of plasma lyso-Gb(3) for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease
por: Bichet, Daniel G., et al.
Publicado: (2020)

Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb(3)) in Fabry Transgenic Mice and in the Plasma of Fabry Patients
por: Young-Gqamana, Brandy, et al.
Publicado: (2013)

Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat: a modified Delphi study
por: Bichet, Daniel G., et al.
Publicado: (2023)

Long-Term Monitoring of Cardiac Involvement under Migalastat Treatment Using Magnetic Resonance Tomography in Fabry Disease
por: Gatterer, Constantin, et al.
Publicado: (2023)

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, et al.
Publicado: (2017)

Migalastat: A Review in Fabry Disease
por: McCafferty, Emma H., et al.
Publicado: (2019)

Correction to: Migalastat: A Review in Fabry Disease
por: McCafferty, Emma H., et al.
Publicado: (2019)

In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
por: Lenders, Malte, et al.
Publicado: (2020)

Medication adherence in Fabry patients treated with migalastat: Real world experience
por: Riccio, Eleonora, et al.
Publicado: (2023)

Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice
por: Wu, Yi Shuan, et al.
Publicado: (2021)

A Pharmacogenetic Approach to Identify Mutant Forms of α-Galactosidase A that Respond to a Pharmacological Chaperone for Fabry Disease
por: Wu, Xiaoyang, et al.
Publicado: (2011)

Impact of enzyme replacement therapy and migalastat on left atrial strain and cardiomyopathy in patients with Fabry disease
por: Pogoda, Christian, et al.
Publicado: (2023)

Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological Chaperone Migalastat
por: Braunstein, Hila, et al.
Publicado: (2020)

Response to “Oral Chaperone Therapy Migalastat for the Treatment of Fabry Disease: Potentials and Pitfalls of Real‐World Data”
por: Müntze, Jonas, et al.
Publicado: (2019)

Biomarkers for Monitoring Renal Damage Due to Fabry Disease in Patients Treated with Migalastat: A Review for Nephrologists
por: Jaurretche, Sebastián, et al.
Publicado: (2022)

Pulsed oral sirolimus in advanced autosomal-dominant polycystic kidney disease (Vienna RAP Study): study protocol for a randomized controlled trial
por: Riegersperger, Markus, et al.
Publicado: (2015)

Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α
por: Kishnani, Priya, et al.
Publicado: (2017)

Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data
por: Riccio, Eleonora, et al.
Publicado: (2020)

Migalastat Treatment in a Kidney-Transplanted Patient with Fabry Disease and N215S Mutation: The First Case Report
por: Di Stefano, Valeria, et al.
Publicado: (2021)

Gaucher Disease in Bone: From Pathophysiology to Practice
por: Hughes, Derralynn, et al.
Publicado: (2019)

Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)
por: Hughes, Derralynn A., et al.
Publicado: (2022)

Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on “Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158”
por: Hughes, Derralynn A., et al.
Publicado: (2023)

Oral Chaperone Therapy Migalastat for Treating Fabry Disease: Enzymatic Response and Serum Biomarker Changes After 1 Year
por: Müntze, Jonas, et al.
Publicado: (2019)

The GALA project: practical recommendations for the use of migalastat in clinical practice on the basis of a structured survey among Italian experts
por: Chimenti, Cristina, et al.
Publicado: (2020)

Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?
por: Perretta, Fernando, et al.
Publicado: (2023)

An updated classification of thrombotic microangiopathies and treatment of complement gene variant-mediated thrombotic microangiopathy
por: Aigner, Christof, et al.
Publicado: (2019)

Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects
por: Ino, Hiroko, et al.
Publicado: (2013)

Randomized, Single Blind, Controlled Trial to Evaluate the Prime-Boost Strategy for Pneumococcal Vaccination in Renal Transplant Recipients
por: Tobudic, Selma, et al.
Publicado: (2012)

First case of atypical haemolytic uraemic syndrome following COVID-19 vaccination with BNT162b2
por: Schmidt, Sophie H, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_a8jrfsh2h25sodtbik1m47dmeq