Search Results - "Karunakara, Ajith"

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  1. 1
    The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis

    The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis by Lang, Frederick M., Korner, Paul, Harnett, Mark, Karunakara, Ajith, Tifft, Cynthia J.

    Published in Molecular genetics and metabolism (01-03-2020)
    “…Type 1 GM1 gangliosidosis is an ultra-rare, rapidly fatal lysosomal storage disorder, with life expectancy of <3 years of age. To date, only one prospective…”
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  2. 2
    DNL310 normalizes primary storage substrates, corrects biomarkers of lysosomal dysfunction and reduces biomarkers of neuronal injury (neurofilament light chain) in MPS II: 2-year interim analysis of a phase 1/2 study

    DNL310 normalizes primary storage substrates, corrects biomarkers of lysosomal dysfunction and reduces biomarkers of neuronal injury (neurofilament light chain) in MPS II: 2-year interim analysis of a phase 1/2 study by Bhalla, Akhil, Tsogtbaatar, Khishig, Tran, Chau, Ravi, Ritesh, Fang, Meng, Zubizarreta, Imanol, Caprihan, Rupa, Karunakara, Ajith, Zhu, Yuda, Sinclair, Elizabeth, Jacquemet-Engelhart, Dominique, Kast, Johannes, Hung, Tony, Scearce-Levie, Kimberly, Troyer, Matt, Chin, Peter, Meirelles, Katia, Bakardjiev, Anna

    Published in Molecular genetics and metabolism (01-02-2024)
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  3. 3
    DNL310 normalizes primary storage substrates and biomarkers of lysosomal dysfunction in neuronopathic MPS II: 2-year interim analysis of a phase 1/2 study

    DNL310 normalizes primary storage substrates and biomarkers of lysosomal dysfunction in neuronopathic MPS II: 2-year interim analysis of a phase 1/2 study by Bhalla, Akhil, Tsogtbaatar, Khishig, Ravi, Ritesh, Fang, Meng, Davis, Sonnet S., Zubizarreta, Imanol, Karunakara, Ajith, Zhu, Yuda, Sinclair, Elizabeth, Jacquemet-Engelhart, Dominique, Dhuria, Shyeilla V., Hung, Tony, Troyer, Matthew, Chin, Peter, Harris, Jeff M., Bakardjiev, Anna

    Published in Molecular genetics and metabolism (01-02-2023)
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  4. 4
    eP259: A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis

    eP259: A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis by Tift, Cynthia, D'Souza, Precilla, Johnston, Jean, Acosta, Maria, Nicoli, Elena-Raluca, Rothermel, Caroline, Thurm, Audrey, Karunakara, Ajith, Thorp, Benjamin, Ross, Peter, Jameson, John, Sheehan, Michael, Vaughn, Toby, Valencia, Donna, De Boever, Erika

    Published in Genetics in medicine (01-03-2022)
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  5. 5
    Phase 1/2 trial of AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and juvenile-onset GM1 gangliosidosis

    Phase 1/2 trial of AXO-AAV-GM1 (AAV9-GLB1) gene therapy for infantile- and juvenile-onset GM1 gangliosidosis by Tifft, Cynthia, D’Souza, Precilla, Johnston, Jean M., Acosta, Maria T., Nicoli, Elena-Raluca, Rothermel, Caroline, Thurm, Audrey, Karunakara, Ajith, Thorp, Benjamin, Ross, Peter, Jameson, John, Sheehan, Michael, Vaughn, Toby, Valencia, Donna, De Boever, Erika, Corcoran, Gavin

    Published in Molecular genetics and metabolism (01-02-2022)
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