13 results on '"Morell, A."'
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2. Stroke characterisation and description of identified risk factors in a Fabry disease large cohort.
3. Fabry Outcome Survey (FOS): Highlights from a 20-year patient registry of Fabry disease.
4. Chronic immune thrombocytopenia refractory to treatment in a patient with Gaucher disease with a common genotype.
5. Amyloid deposition in a patient with the complex Gaucher disease/Parkinson disease.
6. Extracellular vesicles increase the enzymatic activity of lysosomal proteins and improve the efficacy of enzyme replacement therapy in Fabry disease.
7. Targeted nanoliposomes for the treatment of Fabry disease.
8. Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year Fabry Outcome Survey analysis.
9. Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events.
10. Ophthalmological manifestations in Fabry disease children in the Fabry Outcome Survey.
11. Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis.
12. Increase in left ventricular mass index and acroparesthesia incidence in children with Fabry disease correlates with their GLA mutation
13. Measuring patient experiences in fabry disease: validation of the Fabry Outcome Survey (FOS) paediatric health and pain questionnaire
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