Vote: Rapid Fire Session
Rapid Fire Scoring
Most Inspiring Presentation
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Select One
Melissa A. Calton: Non-clinical evaluation of 4D-310 in combination with rituximab/sirolimus
Dawn A. Laney: Development and validation of an automated predictive scoring system to identify patients at increased risk for Fabry disease using Japanese electronic cardiac failure data
Ashish Gupta: Long term enzyme replacement therapy after hematopoietic stem cell transplant results in immune tolerance and improved biochemical outcomes
Slawomir Wantuch: Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease
Jennifer Goldstein: Pseudodeficiency: A poorly defined and misunderstood term in an era of precision medicine
Merve Emecen Sanli: Gene replacement therapy for MPS IIIC with AAV9/HGSNAT vector
Karen Bean: Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD)
Arndt Rolfs: Genetic stratification for Parkinson disease subjects for future personalized trails and therapies – Sidransky syndrome a new entity
Michael H. Gelb: Massively parallel biochemical annotation of VOUS for lysosomal disorders
Most Debated Presentation
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Select One
Melissa A. Calton: Non-clinical evaluation of 4D-310 in combination with rituximab/sirolimus
Dawn A. Laney: Development and validation of an automated predictive scoring system to identify patients at increased risk for Fabry disease using Japanese electronic cardiac failure data
Ashish Gupta: Long term enzyme replacement therapy after hematopoietic stem cell transplant results in immune tolerance and improved biochemical outcomes
Slawomir Wantuch: Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease
Jennifer Goldstein: Pseudodeficiency: A poorly defined and misunderstood term in an era of precision medicine
Merve Emecen Sanli: Gene replacement therapy for MPS IIIC with AAV9/HGSNAT vector
Karen Bean: Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD)
Arndt Rolfs: Genetic stratification for Parkinson disease subjects for future personalized trails and therapies – Sidransky syndrome a new entity
Michael H. Gelb: Massively parallel biochemical annotation of VOUS for lysosomal disorders
Most Thought-Provoking Presentation
Select One
Melissa A. Calton: Non-clinical evaluation of 4D-310 in combination with rituximab/sirolimus
Dawn A. Laney: Development and validation of an automated predictive scoring system to identify patients at increased risk for Fabry disease using Japanese electronic cardiac failure data
Ashish Gupta: Long term enzyme replacement therapy after hematopoietic stem cell transplant results in immune tolerance and improved biochemical outcomes
Slawomir Wantuch: Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease
Jennifer Goldstein: Pseudodeficiency: A poorly defined and misunderstood term in an era of precision medicine
Merve Emecen Sanli: Gene replacement therapy for MPS IIIC with AAV9/HGSNAT vector
Karen Bean: Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD)
Arndt Rolfs: Genetic stratification for Parkinson disease subjects for future personalized trails and therapies – Sidransky syndrome a new entity
Michael H. Gelb: Massively parallel biochemical annotation of VOUS for lysosomal disorders
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