EVALUATION FORM

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Write a number from 1 (worst) to 5 (best) in the box below each item according to your degree of satisfaction.

Venue

Hotel Accomodation

Meals

Social Program

Course Duration

Time Given for Presentations

Time Given for Discussions

Scientific Interaction with Speakers

Simultaneous Translation

Case Reports Session

Material Provided to the Students

Overall Course Organization

Communication before the Meeting

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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

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2 - Pathophysiology of LSDs – Raphael Schiffmann

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3 - Clinical Overview of LSDs - Gregory Pastores

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4 - Genetic Aspects and Molecular Diagnosis of LSDs – Luis Figuera

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Session 2: Gaucher Disease – Monday - Oct 17 (afternoon)

1 - Clinical Aspects of Gaucher Disease – Gregory Pastores

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2 - Biochemical and genetic diagnosis of Gaucher Disease 
      Paula Rozenfeld 

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3 - Gaucher Disease: Management and Follow-up -  Gregory Pastores

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Session 3: Pompe Disease – Monday- Oct 17 (afternoon)

1 - Pompe Disease: Clinical Aspects,  Management and Follow up - Hernan Amartino

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2 - Biochemical and Genetic Diagnosis of Pompe Disease

Maira Burin & Francyne Kubaski

 

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Session 5: Fabry Disease – Tuesday Oct 18 (morning)

1 - Fabry Disease: Clinical Aspects,   Management and Follow up

Raphael Schiffmann 

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2 -Biochemical and Genetic Diagnosis of Fabry Disease 

Maira Burin & Francyne Kubaski

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Session 6: Gangliosidosis – Tuesday Oct 18 (morning)

1 - GM1 and GM2 Gangliosidosis: Clinical Aspects and Management - Hernan Amartino

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2 - Gene Therapy for GM1 gangliosidosis and other neurodegenerative diseases - David Weinstein

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Session 7: Mucopolysaccharidoses – Tuesday Oct 18 (Afternoon)

1 - Clinical Aspects of MPS - Joseph Muenzer

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2 - Alpha-Mannosidosis and Glycoproteinoses Joseph Muenzer

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3 - Biochemical and Genetic Diagnosis of MPS and Glycoproteinoses -          Maira Burin & Francyne Kubaski 

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4- Treatment for MPS: Approved Therapies - Roberto Giugliani

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5 - Treatment for MPS: New Therapies - Joseph Muenzer

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Session 8: Other LSDs – Wednesday - Oct 19 (Morning)

1 - Niemann-Pick type C and Defects of Lysosomal Membrane

Marie Vanier

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2 - Neurodegeneration and Leukodystrophies - Hernan Amartino

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3 - Acid Sphingomyelinase Deficiency: Clinical Aspects and

Management - Marie Vanier

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4 - Newborn screening for LSDs - Roberto Giugliani

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Session 10: Wrap‐Up – Thursday - Oct 20 (Afternoon)

1 - Inflammation and LSDs - Paula Rozenfeld

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2 - Hematopoietic Stem Cell Transplantation in LSDs - Carmem Bonfim

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3 - Diagnosis of LSDs in the Genomic Era - Filippo Vairo

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4 - Genome Editing - Guilherme Baldo

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5 - Gene Therapy and Future Prospects - Raphael Schiffmann

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