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Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease

Autor: Kevin Hammon, Greg deHart, Brian R. Vuillemenot, Derek Kennedy, Don Musson, Charles A. O’Neill, Martin L. Katz, Joshua W. Henshaw

Publikováno v: Clinical and Translational Science, Vol 14, Iss 5, Pp 1810-1821 (2021)

Abstract Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is an ultra‐rare pediatric neurodegenerative disorder characterized by deficiency of the lysosomal enzyme tripeptidyl peptidase‐1 (TPP1). In the absence of adequate TPP1, lysosomal sto

Externí odkaz: https://doaj.org/article/0b3f82eee32841929b398938b7d967e2

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Dose selection for intracerebroventricular cerliponase alfa in children with CLN2 disease, translation from animal to human in a rare genetic disease

Autor: Martin L. Katz, Brian R. Vuillemenot, Greg de Hart, Don Musson, Joshua Henshaw, Charles A. O'Neill, Derek Kennedy, Kevin Hammon

Publikováno v: Clinical and Translational Science
Clinical and Translational Science, Vol 14, Iss 5, Pp 1810-1821 (2021)

Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is an ultra‐rare pediatric neurodegenerative disorder characterized by deficiency of the lysosomal enzyme tripeptidyl peptidase‐1 (TPP1). In the absence of adequate TPP1, lysosomal storage mate

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::28b9f7df12c1312cc9482204d3472724
https://pubmed.ncbi.nlm.nih.gov/34076336

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Abstract A220: Inhibition of PBMC PARP activity with the novel PARP 1/2 inhibitor BMN 673 in patients with advanced solid tumors

Autor: Andrew Dorr, Johann S. de Bono, Huiyu Zhou, Nicola J. Curtin, Lida Mina, Joshua Henshaw, Evelyn Wang, Ashleigh Herriott, Don Musson, Ramesh K. Ramanathan, Charles A. O'Neill, Miranda J. Patterson

Publikováno v: Molecular Cancer Therapeutics. 12:A220-A220

Introduction: BMN 673 is a novel, potent (IC50 < 1.0 nM) inhibitor of Poly(ADP-ribose) polymerase (PARP) 1 and PARP 2 in clinical development for the treatment of genetically defined cancers. Methods: The pharmacokinetics (PK) and pharmacodynamics (P

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::f18970d916240e225ca5f1008a3fc823
https://doi.org/10.1158/1535-7163.targ-13-a220

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Abstract B67: Correlation of pharmacokinetics (PK), pharmacodynamics (PD) and in vivo antitumor activity of BMN 673 in preclinical models

Autor: Don Musson, Ying Feng, Oriane Scholler, Huiyu Zhou, Yuqiao Shen, Leonard Post

Publikováno v: Molecular Cancer Therapeutics. 10:B67-B67

Poly (ADP-ribose) polymerase (PARP) inhibitors are emerging as an exciting class of novel chemotherapeutic agents for the treatment of cancers with specific DNA-repair defects, such as homologous recombination (HR) deficiency as a result of mutations

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::3071c7bd443a6581a08eb714ac1c27de
https://doi.org/10.1158/1535-7163.targ-11-b67

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