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Case Reports Session
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1 - GENE THERAPY FOR LDs - Raphael Schiffmann
2 - GENOME EDITING AND NOVEL APPROACHES FOR THE TREATMENT OF LDS - Natalia Gomez-Ospina
3 - LDS DIAGNOSIS AND THERAPY LANDSCAPE / KEY TAKEWAYS / CLOSURE - Roberto Giugliani
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Scientific Interaction with Speakers
EVALUATION OF SCIENTIFIC ACTIVITIES
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1 - CELL BIOLOGY AND BIOCHEMISTRY OF LYSOSOMES - Guilherme Baldo
2 - PATHOPHYSIOLOGY OF LDS - Carlos E. Prada
3 - CLINICAL OVERVIEW AND GENETIC ASPECTS OF LDs -
Luis Figuera
1 - MECHANISM OF NEURODEGENERATION IN THE LDs -Hernan Amartino
2 - CLINICAL ASPECTS AND MANAGEMENT OF GAUCHER DISEASE, ASMD, AND CESD
Gregory Pastores
3 - BIOCHEMICAL AND GENETIC DIAGNOSIS OF GAUCHER DISEASE
Paula Rozenfeld
4 - CLINICAL ASPECTS OF MPS AND RELATED DISEASES -Joseph Muenzer
5 - BIOCHEMICAL AND GENETIC DIAGNOSIS OF MPS AND MUCOLIPIDOSES
Francyne Kubaski
6 - TREATMENT FOR MPS AND RELATED DISEASES: APPROVED THERAPIES AND NEW DEVELOPMENTS
Joseph Muenzer
1 - GM1 AND GM2 GANGLIOSIDOSES
Hernan Amartino
2 - CLINICAL ASPECTS AND MANAGEMENT OF POMPE DISEASE - Uma Ramaswami
3 - BIOCHEMICAL AND GENETIC DIAGNOSIS ASMD AND POMPE DISEASE
4 - LYSOSOMAL LEUKODYSTROPHIES -
Marie Vanier
5 - CLINICAL ASPECTS AND MANAGEMENT OF FABRY DISEASE -
Raphael Schiffmann
6 - BIOCHEMICAL AND GENETIC DIAGNOSIS OF FABRY DISEASE -
Uma Ramaswami
7 - HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR LDS - Natalia Gomez-Ospina
8 - VARIANT INTERPRETATION GUIDELINES FOR LDS -
Filippo Vairo
9 - DRUG DEVELOPMENT PROCESS FROM THE BENCH TO THE PHARMACY - Guilherme Baldo
1 - NIEMANN PICK C DISEASE - Marie Vanier
2 - OVERVIEW OF SELECTED LESS FREQUENT LDS (DANON, CYSTINOSIS, FARBER, ETC.) - Gregory Pastores​
3 - INFLAMMATION AND LDs - Paula Rozenfeld
4 - NEONATAL SCREENING FOR LDs - Carlos E. Prada
5 - DIAGNOSIS OF LDS IN THE MULTI-OMIC ERA​ -
6 - KEY SKILLS AND INSIGHTS FOR EMERGING LD SPECIALISTS
Uma Ramaswami, Gregory Pastores, Joseph Muenzer,
Raphael Schiffmann, Marie Vanier
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