Vote: Rapid Fire Session
Rapid Fire Scoring
Most Inspiring Presentation
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Select One
Karolina M. Stepien: Evidence of secondary mitochondrial dysfunction in alpha-mannosidosis
Giuseppe Uras: Neuronal dysfunction beyond lysosomes in Fabry disease: Evidence from iPSC-derived forebrain neurons
Betul Celik: Co-transplantation of hematopoietic stem cells and highly purified rapidly expanding clones (REC) of human mesenchymal stem cells rescued the bone pathology of MPS IVA mice
Ryunosuke Sanada: Development of gene editing technologies to correct a mutation in GNPTAB of mucolipidosis type II/III patients
Maria Ester Bernardo: Sustained supraphysiological alpha-L-iduronidase (IDUA) activity, reduction of glycosaminoglycans (GAGs)...
Tahseen Mozaffar: 208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers
Kathy E. Meyer: A combined fertility, embryofetal development, AAV integration and germline transmission risk study in mice with isaralgagene civaparvovec (ST-920) for Fabry disease
Stephan Hold: Breaking barriers in lysosomal disorder screening: A novel simultaneous LC-MS/MS approach for Tay-Sachs, Sandhoff, and GM1 gangliosidosis diseases
Sam Hopkins: Preclinical and clinical safety and activity of AB-1009: A new AAV gene therapy for Pompe disease
Boris Sevarika: Nanotechnology-enabled enzyme replacement therapy: Delivery enhancement and immunogenicity elimination through encapsulation
Magnar Bjørås: Nizubaglustat reinstates pro-neuronal transcriptional programs in human CLN3 retinal organoids
Joseph Skeate: Engineered T cell micropharmacies as a platform for in vivo enzyme replacement therapy in lysosomal disorders
Most Debated Presentation
*
Select One
Karolina M. Stepien: Evidence of secondary mitochondrial dysfunction in alpha-mannosidosis
Giuseppe Uras: Neuronal dysfunction beyond lysosomes in Fabry disease: Evidence from iPSC-derived forebrain neurons
Betul Celik: Co-transplantation of hematopoietic stem cells and highly purified rapidly expanding clones (REC) of human mesenchymal stem cells rescued the bone pathology of MPS IVA mice
Ryunosuke Sanada: Development of gene editing technologies to correct a mutation in GNPTAB of mucolipidosis type II/III patients
Maria Ester Bernardo: Sustained supraphysiological alpha-L-iduronidase (IDUA) activity, reduction of glycosaminoglycans (GAGs)...
Tahseen Mozaffar: 208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers
Kathy E. Meyer: A combined fertility, embryofetal development, AAV integration and germline transmission risk study in mice with isaralgagene civaparvovec (ST-920) for Fabry disease
Stephan Hold: Breaking barriers in lysosomal disorder screening: A novel simultaneous LC-MS/MS approach for Tay-Sachs, Sandhoff, and GM1 gangliosidosis diseases
Sam Hopkins: Preclinical and clinical safety and activity of AB-1009: A new AAV gene therapy for Pompe disease
Boris Sevarika: Nanotechnology-enabled enzyme replacement therapy: Delivery enhancement and immunogenicity elimination through encapsulation
Magnar Bjørås: Nizubaglustat reinstates pro-neuronal transcriptional programs in human CLN3 retinal organoids
Joseph Skeate: Engineered T cell micropharmacies as a platform for in vivo enzyme replacement therapy in lysosomal disorders
Most Thought-Provoking Presentation
*
Select One
Karolina M. Stepien: Evidence of secondary mitochondrial dysfunction in alpha-mannosidosis
Giuseppe Uras: Neuronal dysfunction beyond lysosomes in Fabry disease: Evidence from iPSC-derived forebrain neurons
Betul Celik: Co-transplantation of hematopoietic stem cells and highly purified rapidly expanding clones (REC) of human mesenchymal stem cells rescued the bone pathology of MPS IVA mice
Ryunosuke Sanada: Development of gene editing technologies to correct a mutation in GNPTAB of mucolipidosis type II/III patients
Maria Ester Bernardo: Sustained supraphysiological alpha-L-iduronidase (IDUA) activity, reduction of glycosaminoglycans (GAGs)...
Tahseen Mozaffar: 208-week outcomes of cipaglucosidase alfa plus miglustat in patients with late-onset Pompe disease treated from PROPEL baseline: Muscle function and biomarkers
Kathy E. Meyer: A combined fertility, embryofetal development, AAV integration and germline transmission risk study in mice with isaralgagene civaparvovec (ST-920) for Fabry disease
Stephan Hold: Breaking barriers in lysosomal disorder screening: A novel simultaneous LC-MS/MS approach for Tay-Sachs, Sandhoff, and GM1 gangliosidosis diseases
Sam Hopkins: Preclinical and clinical safety and activity of AB-1009: A new AAV gene therapy for Pompe disease
Boris Sevarika: Nanotechnology-enabled enzyme replacement therapy: Delivery enhancement and immunogenicity elimination through encapsulation
Magnar Bjørås: Nizubaglustat reinstates pro-neuronal transcriptional programs in human CLN3 retinal organoids
Joseph Skeate: Engineered T cell micropharmacies as a platform for in vivo enzyme replacement therapy in lysosomal disorders
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