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Pilar Alfonso Selected Research

miglustat (Zavesca)

1/2018Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
12/2011Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase.
12/2009Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project.
7/2007Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination.
5/2006Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher's disease.
9/2005Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glucosidase in cells transfected with several Gaucher disease mutations.

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Pilar Alfonso Research Topics

Disease

20Gaucher Disease (Gaucher's Disease)
12/2020 - 05/2003
1Type C Niemann-Pick Disease (Niemann Pick Disease, Type C)
03/2016
1Globoid Cell Leukodystrophy (Krabbe Disease)
02/2016
1Fabry Disease (Fabry's Disease)
02/2016
1Pain (Aches)
01/2011
1Myoclonic Epilepsies (Myoclonic Encephalopathy)
07/2007
1Dystonia (Limb Dystonia)
07/2007
1Seizures (Absence Seizure)
07/2007

Drug/Important Bio-Agent (IBA)

10Glucosylceramidase (Glucocerebrosidase)IBA
10/2013 - 05/2003
6miglustat (Zavesca)FDA Link
01/2018 - 09/2005
5EnzymesIBA
12/2020 - 05/2003
4ceramide glucosyltransferaseIBA
03/2016 - 05/2006
3imigluceraseFDA Link
12/2011 - 07/2007
2GlycosphingolipidsIBA
02/2016 - 12/2011
1Cytochrome P-450 CYP2D6 (CYP2D6)IBA
12/2020
1CytochromesIBA
12/2020
1CholesterolIBA
03/2016
1sphingosyl beta-glucosideIBA
02/2016
1Psychosine (Galactosylsphingosine)IBA
02/2016
1Protein Isoforms (Isoforms)IBA
07/2006
1chitotriosidaseIBA
07/2006
1Biomarkers (Surrogate Marker)IBA
07/2006
1Proteins (Proteins, Gene)FDA Link
07/2006
1Glucosylceramides (Glucocerebrosides)IBA
09/2005
1LipidsIBA
05/2003
1AcidsIBA
05/2003

Therapy/Procedure

4Enzyme Replacement Therapy
01/2018 - 05/2003
4Therapeutics
01/2017 - 07/2007
1Ambulatory Care (Outpatient Care)
12/2009