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Polo-like kinase inhibitors increase AAV production by halting cell cycle progression Fisher, Kaylin; Grafton, Francis; Ispaso, Francesca; Tworig, Joshua; Derler, Rupert; Sonntag, Florian; Hoerer, Markus; Schulze, Andreas; Reid, Christopher A.; Mandegar, Mohammad A. Share Save
Neonatal Encephalopathy Ambrose, Anastasia; Mcniven, Vanda; Wilson, Diane; Tempes, Aleksandra; Underwood, Mary; Chau, Vann; Schulze, Andreas; Wyszynska, Agnieszka; Desch, Karl; Malik, Anna R.; Mercimek-Andrews, Saadet Share Save
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Unraveling the Link: Seizure Characteristics and Ammonia Levels in Urea Cycle Disorder During Hyperammonemic Crises Chanvanichtrakool, Mongkol; Schreiber, John M.; Chen, Wei-Liang; Barber, John; Zhang, Anqing; Mew, Nicholas Ah; Schulze, Andreas; Wilkening, Greta; Nagamani, Sandesh C. S.; Gropman, Andrea Share Save
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
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Arrested development of oligodendrocytes by guanidino compounds, not arginine, fosters dysmyelination in arginase deficiency Diep, Taryn; Zhang, Lizzie; Le, Colleen; Liu, Xiao-Bo; Patel, Puja; Bakshi, Stuti; Tkachyova, Ilona; Schulze, Andreas; Lipshutz, Gerald S. 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
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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Potent broadly neutralizing antibody VIR-3434 controls hepatitis B and D virus infection and reduces HBsAg in humanized mice Lempp, Florian A.; Volz, Tassilo; Cameroni, Elisabetta; Benigni, Fabio; Zhou, Jiayi; Rosen, Laura E.; Noack, Julia; Zatta, Fabrizia; Kaiser, Hannah; Bianchi, Siro; Lombardo, Gloria; Jaconi, Stefano; Vincenzetti, Lucia; Imam, Hasan; Soriaga, Leah B.; Passini, Nadia; Belnap, David M.; Schulze, Andreas; Luetgehetmann, Marc; Telenti, Amalio; Cathcart, Andrea L.; Snell, Gyorgy; Purcell, Lisa A.; Hebner, Christy M.; Urban, Stephan; Dandri, Maura; Corti, Davide; Schmid, Michael A. Share Save
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Biallelic loss-of-function variants in RABGAP1 cause a novel neurodevelopmental syndrome Oh, Rachel Youjin; Deshwar, Ashish R.; Marwaha, Ashish; Sabha, Nesrin; Tropak, Michael; Hou, Huayun; Yuki, Kyoko E.; Wilson, Michael D.; Rump, Patrick; Lunsing, Roelineke; Elserafy, Noha; Chung, Clara W. T.; Hewson, Stacy; Klein-Rodewald, Tanja; Calzada-Wack, Julia; Sanz-Moreno, Adrian; Kraiger, Markus; Marschall, Susan; Fuchs, Helmut; Gailus-Durner, Valerie; de Angelis, Martin Hrabe; Dowling, James; Schulze, Andreas Share Save
X-linked creatine transporter deficiency results in prolonged QTc and increased sudden death risk in humans and disease model Levin, Mark D.; Bianconi, Simona; Smith, Andrew; Cawley, Niamh X.; Do, An Dang; Hammond, Dylan; Grafstein, Julia F.; Thurm, Audrey; Miller, Judith; Perreault, John; Noguchi, Audrey; Springer, Danielle; Kozel, Beth A.; Spurney, Christopher F.; Wassif, Christopher A.; Yu, Zu-Xi; Schulze, Andreas; Porter, Forbes D.; Hannah-Shmouni, Fady Share Save
Core Outcome Sets for Medium-Chain Acyl-CoA Dehydrogenase Deficiency and Phenylketonuria Pugliese, Michael; Tingley, Kylie; Chow, Andrea; Pallone, Nicole; Smith, Maureen; Chakraborty, Pranesh; Geraghty, Michael T.; Irwin, Julie K.; Mitchell, John J.; Stockler, Sylvia; Nicholls, Stuart G.; Offringa, Martin; Rahman, Alvi; Tessier, Laure A.; Butcher, Nancy J.; Iverson, Ryan; Lamoureux, Monica; Clifford, Tammy J.; Hutton, Brian; Paik, Karen; Tao, Jessica; Skidmore, Becky; Coyle, Doug; Duddy, Kathleen; Dyack, Sarah; Greenberg, Cheryl R.; Jain Ghai, Shailly; Karp, Natalya; Korngut, Lawrence; Kronick, Jonathan; MacKenzie, Alex; MacKenzie, Jennifer; Maranda, Bruno; Potter, Murray; Prasad, Chitra; Schulze, Andreas; Sparkes, Rebecca; Taljaard, Monica; Trakadis, Yannis; Walia, Jagdeep; Potter, Beth K. Share Save
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