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Your search keyword '"Truxal, Kristen"' showing total 3 results
Start Over Author "Truxal, Kristen" Topic gene therapy
3 results on '"Truxal, Kristen"'

1. Interim results of Transpher A, a multicenter, single-dose clinical trial of UX111 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA).

Author

Flanigan, Kevin M., Smith, Nicholas, Couce, Maria Luz, Rajan, Deepa, Truxal, Kristen, McBride, Kim L., de Castro Lopez, Maria Jose, Fuller, Maria, Taylor, John, Del Campo, Ana B., Grachev, Igor, and Lau, Heather

Subjects
*SANFILIPPO syndrome, *GENE therapy, *MUCOPOLYSACCHARIDOSIS, *CLINICAL trials
Published
2023
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2. Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA).

Author

Flanigan, Kevin M., Smith, Nicholas J.C., Couce, Maria Luz, Truxal, Kristen V., McBride, Kim L., Simmons, Tabatha, de Castro, Maria Jose, Cope, Krista, Oreiro, Maria Teresa, Jaensch, Louise, Fuller, Maria, and Ruiz, Juan

Subjects
*GENE therapy, *SANFILIPPO syndrome, *CLINICAL trials, *HEPARAN sulfate, *INTRAVENOUS therapy
Published
2020
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3. Safety, tolerability and preliminary evidence of biopotency in Transpher B, a multicenter, single-dose, phase 1/2 clinical trial of ABO-101 gene therapy for Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB).

Author

McBride, Kim L., Couce, Maria Luz, Flanigan, Kevin M., Truxal, Kristen V., Simmons, Tabatha, de Castro, Maria Jose, Rinaldi, Federica, Oreiro, Maria Teresa, Fuller, Maria, and Ruiz, Juan

Subjects
*GENE therapy, *SANFILIPPO syndrome, *CLINICAL trials, *HEPARAN sulfate, *INTRAVENOUS therapy
Published
2020
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