RGX-121 gene therapy for severe Mucopolysaccharidosis Type II (MPS II): Interim neurodevelopmental outcomes and biomarker results

被引：0

作者：

Harmatz, Paul ^{[1
]}

Escolar, Maria ^{[2
]}

Ficicioglu, Can ^{[3
]}

Giugliani, Roberto ^{[4
]}

Cho, Yoonjin ^{[5
]}

Phillips, Dawn ^{[5
]}

Nevoret, Marie-Laure ^{[5
]}

Falabella, Paulo ^{[5
]}

机构：

[1] UCSF Benioff Childrens Hosp Oakland, Gastroenterol, Oakland, CA USA

[2] UPMC Childrens Hosp Pittsburgh, Pediat, Pittsburgh, PA USA

[3] Childrens Hosp Philadelphia, Pediat, Philadelphia, PA 19104 USA

[4] Univ Fed Rio Grande do Sul, Med Genet, Porto Alegre, RS, Brazil

[5] REGENXBIO, Rockville, MD USA

来源：

NEUROLOGY | 2021年 / 96卷 / 15期

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

4164

引用

页数：4

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[31] Mucopolysaccharidosis type II (MPS II): Evaluate of food intakes in patients on enzyme replacement therapy [J].

Oliveira, R. B. ;

Frangipani, B. J. ;

Lauandos, J. R. ;

Micheletti, C. ;

Silveira, M. T. ;

Vertemati, T. ;

Martins, A. M. .

JOURNAL OF INHERITED METABOLIC DISEASE, 2008, 31 :100-100

[32] Summary of nonclinical data for gene therapy developmental candidate HMI-203 for mucopolysaccharidosis type II (MPS II, or Hunter syndrome) [J].

Smith, Laura J. ;

Patel, Kruti ;

Seabrook, Tania ;

Schulman, Lindsay ;

Zhivich, Victor ;

Kivaa, Monicah ;

Behmoiras, Liana ;

Tzianabos, Alec ;

Faulkner, Deiby ;

Sengooba, Arnold ;

Avila, Nancy ;

Newman, Jennifer ;

Zhao, Jingyan ;

Woodcock, Steve ;

Klem, Thomas ;

Smith, Sean ;

Seymour, Albert ;

Francone, Omar ;

Gingras, Jacinthe .

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[33] Validation of a GMP stem cell gene therapy manufacturing process for mucopolysaccharidosis type II (MPS II) in preparation for an approved phase I/II clinical trial [J].

Ellison, Stuart M. ;

Buckland, Karen ;

Ishikawalearmonth, Yuko ;

Booth, Claire ;

Wynn, Robert ;

Jones, Simon ;

Bigger, Brian .

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[34] Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II (MPS II): A phase 1/2 study [J].

Muenzer, Joseph ;

Burton, Barbara K. ;

Harmatz, Paul ;

Rajan, Deepa ;

Jones, Simon ;

van den Hout, Hannerieke J. M. ;

Mitchell, John J. ;

Troyer, Matt ;

Engmann, Natalie ;

Caprihan, Rupa ;

Bhalla, Akhil ;

Zubizarreta, Imanol ;

Weichert, Arlette ;

Chin, Peter ;

Ho, Carole .

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[35] Long-Term Expression of HMI-203: Investigational Gene Therapy Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome [J].

Smith, Laura ;

Patel, Kruti ;

Gingras, Jacinthe ;

Tzianabos, Alec ;

Schulman, Lindsay ;

Zhivich, Victor ;

Kivaa, Monicah ;

Faulkner, Deiby ;

Sengooba, Arnold ;

Behmoiras, Liana ;

Seabrook, Tania ;

Dollive, Serena ;

Avila, Nancy ;

Zhao, Jingyan ;

White, Yvonne ;

Newman, Jenn ;

Woodcock, Steve ;

Smith, Sean ;

Francone, Omar ;

Seymour, Albert .

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[36] Disease course after cessation of enzyme replacement therapy (ERT) in 5 patients with type II mucopolysaccharidosis (MPS II) [J].

Sivananthan, Siyamini ;

Stewart, Catherine ;

Finnegan, Niamh ;

Santra, Saikat ;

Vellodi, Ashok ;

Cleary, Maureen ;

Chakrapani, Anupam .

MOLECULAR GENETICS AND METABOLISM, 2015, 114 (02) :S107-S108

[37] Neurodevelopmental outcomes of hematopoietic stem cell transplantation for mucopolysaccharidosis type II: A prospective, longitudinal study [J].

Bascou, Nicholas A. ;

Kiely, Bridget ;

Safonova, Aleksandra ;

Beltran, Maria L. ;

Poe, Michele D. ;

Szabolcs, Paul ;

Escolar, Maria L. .

MOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) :S27-S27

[38] Neurodevelopmental Outcomes of Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis Type II: A Prospective, Longitudinal Study [J].

Escolar, Maria ;

Bascou, Nicholas ;

Kiely, Bridget ;

Safonova, Aleksandra ;

Beltran, Maria ;

Poe, Michele ;

Szabolcs, Paul .

BONE MARROW TRANSPLANTATION, 2020, 55 (SUPPL 1) :437-437

[39] Preclinical Development of an Ex Vivo Gene Therapy for Mucopolysaccharidosis Type II [J].

Poletti, Valentina ;

Das, Sabyasachi ;

Rruga, Fatlum ;

Dimartino, Agnese ;

Montepeloso, Annita ;

Patel, Janki ;

Ferro, Francesca ;

Cavalca, Eleonora ;

Politi, Letterio ;

Biffi, Alessandra .

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[40] Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities [J].

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