European expert consensus statement on therapeutic goals in Fabry disease

C Wanner, M Arad, R Baron, A Burlina, PM Elliott… - Molecular genetics and …, 2018 - Elsevier
Background Fabry disease, an inherited lysosomal storage disorder, causes multi-organ
pathology resulting in substantial morbidity and a reduced life expectancy. Although Fabry …

[HTML][HTML] The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: a systematic literature review by a European panel of …

DP Germain, PM Elliott, B Falissard, VV Fomin… - Molecular genetics and …, 2019 - Elsevier
Background Enzyme replacement therapy (ERT) with recombinant human α-galactosidase
has been available for the treatment of Fabry disease since 2001 in Europe and 2003 in the …

[HTML][HTML] Chaperone therapy in Fabry disease

F Weidemann, A Jovanovic, K Herrmann… - International journal of …, 2022 - mdpi.com
Fabry disease is an X-linked lysosomal multisystem storage disorder induced by a mutation
in the alpha-galactosidase A (GLA) gene. Reduced activity or deficiency of alpha …

[HTML][HTML] Treatment of Fabry's disease with the pharmacologic chaperone migalastat

DP Germain, DA Hughes, K Nicholls… - New england journal …, 2016 - Mass Medical Soc
Background Fabry's disease, an X-linked disorder of lysosomal α-galactosidase deficiency,
leads to substrate accumulation in multiple organs. Migalastat, an oral pharmacologic …

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

DA Hughes, K Nicholls, SP Shankar… - Journal of medical …, 2017 - jmg.bmj.com
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 …

Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry

A Ortiz, A Abiose, DG Bichet, G Cabrera… - Journal of Medical …, 2016 - jmg.bmj.com
Background Agalsidase β is a form of enzyme replacement therapy for Fabry disease, a
genetic disorder characterised by low α-galactosidase A activity, accumulation of …

The second-meal phenomenon in type 2 diabetes

A Jovanovic, J Gerrard, R Taylor - Diabetes care, 2009 - Am Diabetes Assoc
OBJECTIVE In health, the rise in glucose after lunch is less if breakfast is eaten. We
evaluated the second-meal effect in type 2 diabetes. RESEARCH DESIGN AND METHODS …

Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study

DP Germain, E Brand, A Burlina… - Molecular genetics & …, 2018 - Wiley Online Library
Abstract Background The p. Asn215Ser or p. N215S GLA variant has been associated with
late‐onset cardiac variant of Fabry disease. Methods To expand on the scarce phenotype …

External and internal factors affecting the product and business process innovation

N Zakić, A Jovanović, M Stamatović - Facta universitatis-series …, 2008 - bic-pk.ceon.rs
The competitive advantage of a company strongly depends on its possibility to benefit from
innovational activities. Understanding the factors that affect product and process innovation …

The second-meal phenomenon is associated with enhanced muscle glycogen storage in humans

A Jovanovic, E Leverton, B Solanky… - Clinical …, 2009 - portlandpress.com
The rise in blood glucose after lunch is less if breakfast has been eaten. The metabolic basis
of this second-meal phenomenon remains uncertain. We hypothesized that storage of …