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Developing a National Network for Leukodystrophy Research and Care in Canada Chapleau, Alexandra; Le, Adam; Simo, Justin; Venkateswaran, Sunita; Lacaze-Masmonteil, Thierry; Piscopo, Valerio E. C.; Gauthier, Samuel; Villa Tobon, Felipe; Alam, Sabrina Shameen; Lentini, Laura; Brais, Bernard; Ernst, Carl; Mitchell, John J.; Vinh, Donald C.; Zhou, Yang; Kennedy, Timothy E.; Goloff, Naomi; Riham, Badawy; Chapleau, Ron; Smith, Ben; Greger, Valerie; Della Rocca, Josee; Louis, Lynda-Marie; Dike, Ashley; Mcintyre, Lindey L.; Mcintyre, David F.; Tardif, Jerome; Lapointe, Emilie; Barnett, Jennifer; Loignon, Valerie; Bardai, Ghalib; Contant, Sophie; Durcan, Thomas M.; La Piana, Roberta; Bernard, Genevieve Share Save
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Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa) Weinstein, David A.; Derks, Terry G.; Rodriguez-Buritica, David F.; Ahmad, Ayesha; Couce, Maria-Luz; Mitchell, John J.; Riba-Wolman, Rebecca; Mount, Malaya; Sallago, Julieta Bonvin; Ross, Katalin M.; van Der Klauw, Melanie M.; de Boer, Foekje; van Der Schaaf, Caroline; Saavedra, Heather; Martinez-Olmos, Miguel; Atanga, Elvis; Hosseini, Asad; Mitragotri, Deepali; Crombez, Eric Share Save
Long-term outcomes of MPS IVA patients treated with elosulfase alfa: Findings from the Morquio A Registry Study (MARS) after 10 years Mitchell, John J.; Burton, Barbara K.; Campeau, Philippe M.; Ellaway, Carolyn; Guffon, Nathalie; Hughes, Derralynn; Hunt, Abigail; Lail, Alice; Lin, Hsiang-Yu; Lin, Shuan-Pei; Magner, Martin; Reddy, Sheila; Reisewitz, Pascal; Stepien, Karolina M. 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
Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy Rossi, Alessandro; Romano, Roberta; Fecarotta, Simona; Dell'Anno, Margherita; Pecorella, Valentina; Passeggio, Roberta; Zancan, Stefano; Parenti, Giancarlo; Santamaria, Francesca; Borgia, Francesco; Deodato, Federica; Funghini, Silvia; Rupar, Charles A.; Prasad, Chitra; O'Callaghan, Mar; Mitchell, John J.; Valsecchi, Maria Grazia; la Marca, Giancarlo; Galimberti, Stefania; Auricchio, Alberto; Brunetti-Pierri, Nicola Share Save
Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement Howie, Alison H.; Tingley, Kylie; Inbar-Feigenberg, Michal; Mitchell, John J.; Angel, Kim; Gentle, Jenifer; Smith, Maureen; Offringa, Martin; Butcher, Nancy J.; Campeau, Philippe M.; Chakraborty, Pranesh; Chan, Alicia; Fergusson, Dean; Mamak, Eva; McClelland, Peyton; Mercimek-Andrews, Saadet; Mhanni, Aizeddin; Moazin, Zeinab; Rockman-Greenberg, Cheryl; Rupar, C. Anthony; Skidmore, Becky; Stockler, Sylvia; Thavorn, Kednapa; Wyatt, Alexandra; Potter, Beth K. Share Save
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Co-developing longitudinal patient registries for phenylketonuria and mucopolysaccharidoses in Canada Adams, John; Angel, Kim; Mitchell, John J.; Chakraborty, Pranesh; Potter, Beth K.; Inbar-Feigenberg, Michal; Stockler, Sylvia; Lamoureux, Monica; Howie, Alison; Pace, Alex; Butcher, Nancy J.; Rockman-Greenberg, Cheryl; Hayeems, Robin; Laberge, Anne-Marie; Lacaze-Masmonteil, Thierry; Round, Jeff; Offringa, Martin; Oksoui, Maryam; Schulze, Andreas; Speechley, Kathy; Thavorn, Kednapa; Trakadis, Yannis; Wilson, Kumanan Share Save
Family-centred care interventions for children with chronic conditions: A scoping review Chow, Andrea J.; Saad, Ammar; Al-Baldawi, Zobaida; Iverson, Ryan; Skidmore, Becky; Jordan, Isabel; Pallone, Nicole; Smith, Maureen; Chakraborty, Pranesh; Brehaut, Jamie; Cohen, Eyal; Dyack, Sarah; Gillis, Jane; Goobie, Sharan; Greenberg, Cheryl R.; Hayeems, Robin; Hutton, Brian; Inbar-Feigenberg, Michal; Jain-Ghai, Shailly; Khangura, Sara; Mackenzie, Jennifer J.; Mitchell, John J.; Moazin, Zeinab; Nicholls, Stuart G.; Pender, Amy; Prasad, Chitra; Schulze, Andreas; Siriwardena, Komudi; Sparkes, Rebecca N.; Speechley, Kathy N.; Stockler, Sylvia; Taljaard, Monica; Teitelbaum, Mari; Trakadis, Yannis; Van Karnebeek, Clara; Walia, Jagdeep S.; Wilson, Kumanan; Potter, Beth K. Share Save
Interim analysis of a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in mucopolysaccharidosis type II Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Rajan, Deepa; Jones, Simon A.; van den Hout, Hannerieke . J. P.; Mitchell, John; Meirelles, Katia; Engmann, Natalie; Scheller, Adam; Caprihan, Rupa; Bhalla, Akhil; Hung, Tony; Nachtigall, Jason; Zubizarreta, Imanol; Lu, Sam; Zhu, Yuda; Chin, Peter; Troyer, Matt D.; Ho, Carole; Dong, Wei; Model, Fabian Share Save
Somatic outcomes in a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in mucopolysaccharidosis type II Burton, Barbara; Muenzer, Joseph; Harmatz, Paul; Rajan, Deepa; Jones, Simon A.; van den Hout, Hannerieke J. P.; Mitchell, John; Meirelles, Katia; Engmann, Natalie; Scheller, Adam; Caprihan, Rupa; Bhalla, Akhil; Hung, Tony; Nachtigall, Jason; Zubizarreta, Imanol; Lu, Sam; Zhu, Yuda; Chin, Peter; Troyer, Matt D.; Ho, Carole; Dong, Wei; Model, Fabian; Bakardjiev, Anna I. Share Save
Co-developing The Canadian MPS Registry: A longitudinal rare disease patient registry Mitchell, John J.; Inbar-Feigenberg, Michal; Angel, Kim; Chakraborty, Pranesh; Lamoureux, Monica; Adams, John; Potter, Beth K.; Stockler-Ipsirolgu, Sylvia; Howie, Alison H.; Pace, Alex; Butcher, Nancy J.; Greenberg, Cheryl; Hayeems, Robin; Laberge, Anne-Marie; Round, Jeff; Offringa, Martin; Oskoui, Maryam; Ruth, Chelsea; Schulze, Andreas; Speechley, Kathy; Thavorn, Kednapa; Trakadis, Yannis; Wilson, Kumanan; Lacaze, Thierry Share Save
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Development of a core outcome set for mucopolysaccharidoses (MPS) in children: Results from Delphi surveys and a consensus workshop Howie, Alison H.; Wyatt, Alexandra; Inbar-Feigenberg, Michal; Mitchell, John J.; Smith, Maureen; Angel, Kim; Gentle, Jenifer; Butcher, Nancy J.; Offringa, Martin; Campeau, Philippe M.; Chan, Alicia K.; Chakraborty, Pranesh; Chow, Andrea; El Turk, Farah; Greenberg, Cheryl R.; Mamak, Eva; Mhanni, Aziz; Rupar, Tony; Sparkes, Rebecca; Thavorn, Kednapa; Tingley, Kylie; Potter, Beth K. Share Save
Rare diseases and space health: optimizing synergies from scientific questions to care Puscas, Maria; Martineau, Gabrielle; Bhella, Gurjot; Bonnen, Penelope E.; Carr, Phil; Lim, Robyn; Mitchell, John; Osmond, Matthew; Urquieta, Emmanuel; Flamenbaum, Jaime; Iaria, Giuseppe; Joly, Yann; Richer, Etienne; Saary, Joan; Saint-Jacques, David; Buckley, Nicole; Low-Decarie, Etienne Share Save
Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa Mitchell, John J.; Burton, Barbara K.; Bober, Michael B.; Campeau, Philippe M.; Cohen, Shelda; Dosenovic, Sara; Ellaway, Carolyn; Bhattacharya, Kaustuv; Guffon, Nathalie; Hinds, David; Lail, Alice; Lin, Shuan-Pei; Magner, Martin; Raiman, Julian; Schwartz-Sagi, Liat; Stepien, Karolina M. Share Save
Farber disease clinical impact: Patient reported outcomes as a measure of disease burden Mitchell, John; Harmatz, Paul; Selim, Laila; El Din, Iman Gamal; Mungan, Neslihan; Bulut, Fatma; Lampe, Christina; Grant, Christina L.; Ferreira, Carlos R.; Puri, Ratna D.; Bijarnia-Mahay, Sunita; Arslan, Nur; Guelbert, Norberto; Zaki, Maha S.; Kapoor, Seema; Hahn, Andreas; Magnusson, Bo; Sundberg, Erik; Ozen, Seza; Batu, Ezgi D.; Di Rocco, Maja; Gokcay, Gulden; Crosby, Kathleen; Solyom, Alexander Share Save
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