Not logged in Intrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gutierrez-solana, Luis Gonzalez; Ruiz-garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-feigenberg, Michal; Bratkovic, Drago; Rust, Stewart; Hale, Michael; Wu, Yuna; Yee, Karen S.; Whiteman, David A. H.; Alexanderian, David Share Save
An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Rajan, Deepa S.; Jones, Simon A.; van den Hout, Johanna M. P.; Mitchell, John J.; Bhalla, Akhil; Engmann, Natalie J.; Zubizarreta, Imanol; Dong, Wei; Model, Fabian; Watts, Ryan J.; Troyer, Matthew D.; Chin, Peter S.; Ho, Carole Share Save
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Gangliosidoses: Understanding disease evolution in GM1 and GM2 Giugliani, Roberto; Harmatz, Paul; Heron, Benedicte; Hahn, Andreas; Patterson, Marc; Bourchany, Aurelie; Valle, Daniel A. D.; Barone, Rita Maria Elisa; Chabrol, Brigitte; Ardissone, Anna; Batzios, Spyros; Scarpa, Maurizio; Schneider, Susanne A.; Carp, Nicoleta; Freitag, Christian; Gill, Aidan; de Frutos, Laura Lopez Share Save
Campsiite® phase I/II/III: an interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II) Harmatz, Paul; Ficicioglu, Can; Giugliani, Roberto; Rajan, Deepa; Day-Salvatore, Debra-Lynn; Hagood, Joseph; Gilmor, Michelle; Cho, Yoonjin; Mulatya, Caroline; Phillips, Dawn; Boulos, Nidal; Pisani, Laura; Falabella, Paulo Share Save
Application of a natural history study on an investigational drug clinical development Giugliani, Roberto; Valle, Daniel A. D.; Heron, Benedicte; Harmatz, Paul; Hahn, Andreas; Horovitz, Dafne D. G.; Patterson, Marc; Bourchany, Aurelie; Barone, Rita Maria Elisa; Chabrol, Brigitte; Ardissone, Anna; Batzios, Spyros; Scarpa, Maurizio; Schneider, Susanne A.; Carp, Nicoleta; Freitag, Christian; Gill, Aidan; de Frutos, Laura Lopez Share Save
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A phase 1/2 study of LY3884961 (PR001) an AAV9-based gene therapy for Gaucher disease type 2-A clinical update from the PROVIDE trial Neuhaus, Sarah; Tamburri, Paul; Whitley, Chester B.; Jones, Simon A.; Donald, Aimee; Harmatz, Paul; Blair, David R.; Chang, Irene; Gallagher, Renata C.; Rajan, Deepa S.; Goker-Alpan, Ozlem; Beckerman, Yael; Lopez, Victor A.; Hatch, Daniel; Shaughnessy, Lee Share Save
Building safe systems for patients with mucopolysaccharidoses (MPS) - Results of an expert survey on patient safety Lagler, Florian B.; Stepien, Karolina M.; Akyol, Umut; Lampe, Christina; Bruce, Iain A.; Walker, Robert; De Souza, Carolina Fischinger Moura; Mitchell, John J.; Harmatz, Paul; Giugliani, Roberto Share Save
Interim analysis of the efficacy and safety of weekly intravenous tividenofusp alfa in mucopolysaccharidosis type II (MPS II): A phase 1/2 study 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 Share Save
Interim results from a first in human phase 1 clinical trial of in utero enzyme replacement therapy for lysosomal disorders Mackenzie, Tippi C.; Lianoglou, Billie; Canepa, Emma; Herzeg, Akos; Borgas, Beltran; Cohen, Jennifer L.; Bali, Deeksha; Young, Sarah; Gonzalez-Vele, Juan; Munar, Dane; Gelb, Michael H.; Herbs, Zackary (Ari) M.; Chakraborty, Pranesh; Kishnani, Priya; Harmatz, Paul Share Save
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Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life Savarirayan, Ravi; Irving, Melita; Wilcox, William R.; Bacino, Carlos A.; Hoover-Fong, Julie E.; Harmatz, Paul; Polgreen, Lynda E.; Mohnike, Klaus; Prada, Carlos E.; Kubota, Takuo; Arundel, Paul; Leiva-Gea, Antonio; Rowell, Richard; Low, Andrea; Sabir, Ian; Huntsman-Labed, Alice; Day, Jonathan Share Save
CAMPSIITET Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II) Pisani, Laura; Ficicioglu, Can; Harmatz, Paul; Giugliani, Roberto; Rajan, Deepa; Hagood, Joseph; Fiscella, Michele; Yang, Lin; Gilmor, Michelle; Cho, Yoonjin; Mulatya, Caroline; Phillips, Dawn; Boulos, Nidal; Falabella, Paulo Share Save
α- L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial Harmatz, Paul; Giugliani, Roberto; Martins, Ana Maria; Hamazaki, Takashi; Kubo, Toru; Kira, Ryutaro; Minami, Kohtaro; Ikeda, Toshiaki; Moriuchi, Hiroaki; Kawashima, Satoshi; Takasao, Naoko; So, Sairei; Sonoda, Hiroyuki; Hirato, Tohru; Tanizawa, Kazunori; Schmidt, Mathias; Sato, Yuji Share Save
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