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EVALUATION OF SCIENTIFIC ACTIVITIES
(rank 1-worse to 5-best)
Session 1: Introduction to LSDs - Monday – Oct 17 (morning)
1 - Cell Biology and Biochemistry of Lysosomes - Guilherme Baldo
2 - Pathophysiology of LSDs – Raphael Schiffmann
3 - Clinical Overview of LSDs - Gregory Pastores
4 - Genetic Aspects and Molecular Diagnosis of LSDs – Luis Figuera
Session 2: Gaucher Disease – Monday - Oct 17 (afternoon)
1 - Clinical Aspects of Gaucher Disease – Gregory Pastores
2 - Biochemical and genetic diagnosis of Gaucher Disease
Paula Rozenfeld
3 - Gaucher Disease: Management and Follow-up - Gregory Pastores
Session 3: Pompe Disease – Monday- Oct 17 (afternoon)
1 - Pompe Disease: Clinical Aspects, Management and Follow up - Hernan Amartino
2 - Biochemical and Genetic Diagnosis of Pompe Disease
Maira Burin & Francyne Kubaski
Session 5: Fabry Disease – Tuesday Oct 18 (morning)
1 - Fabry Disease: Clinical Aspects, Management and Follow up
Raphael Schiffmann
2 -Biochemical and Genetic Diagnosis of Fabry Disease
Maira Burin & Francyne Kubaski
Session 6: Gangliosidosis – Tuesday Oct 18 (morning)
1 - GM1 and GM2 Gangliosidosis: Clinical Aspects and Management - Hernan Amartino
2 - Gene Therapy for GM1 gangliosidosis and other neurodegenerative diseases - David Weinstein
Session 7: Mucopolysaccharidoses – Tuesday Oct 18 (Afternoon)
1 - Clinical Aspects of MPS - Joseph Muenzer
2 - Alpha-Mannosidosis and Glycoproteinoses - Joseph Muenzer
3 - Biochemical and Genetic Diagnosis of MPS and Glycoproteinoses - Maira Burin & Francyne Kubaski
4- Treatment for MPS: Approved Therapies - Roberto Giugliani
5 - Treatment for MPS: New Therapies - Joseph Muenzer
Session 8: Other LSDs – Wednesday - Oct 19 (Morning)
1 - Niemann-Pick type C and Defects of Lysosomal Membrane
Marie Vanier
2 - Neurodegeneration and Leukodystrophies - Hernan Amartino
3 - Acid Sphingomyelinase Deficiency: Clinical Aspects and
Management - Marie Vanier
4 - Newborn screening for LSDs - Roberto Giugliani
Session 10: Wrap‐Up – Thursday - Oct 20 (Afternoon)
1 - Inflammation and LSDs - Paula Rozenfeld
2 - Hematopoietic Stem Cell Transplantation in LSDs - Carmem Bonfim
3 - Diagnosis of LSDs in the Genomic Era - Filippo Vairo
4 - Genome Editing - Guilherme Baldo
5 - Gene Therapy and Future Prospects - Raphael Schiffmann
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