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SaveShare Save Share Save Characterization of patients with mucopolysaccharidosis type VII (MPS VII) in the disease monitoring program (DMP)
Grant, Christina L.; Wang, Raymond Y.; Giugliani, Roberto; Gonzalez-Meneses, Antonio; Scarpa, Maurizio; Burton, Barbara K.; Portmann, Monica Penon; Oussoren, Esmeralda; Martins, Esmeralda E.; Durand, Consuelo; Chabrol, Brigitte; Hetzer, Joel; Cohen, Jeanne M.; Marsden, Deborah; Merrittii, J. Lawrence Share Save Share Save Sequence variants in HECTD1 result in a variable neurodevelopmental disorderZerafati-Jahromi, Gazelle; Oxman, Elias; Hoang, Hieu D.; Charng, Wu-Lin; Kotla, Tanvitha; Yuan, Weimin; Ishibashi, Keito; Sebaoui, Sonia; Luedtke, Kathryn; Winrow, Bryce; Ganetzky, Rebecca D.; Ruiz, Anna; Manso-Basuz, Carmen; Spataro, Nino; Kannu, Peter; Athey, Taryn; Peroutka, Christina; Barnes, Caitlin; Sidlow, Richard; Anadiotis, George; Magnussen, Kari; Valenzuela, Irene; Moles-Fernandez, Alejandro; Berger, Seth; Grant, Christina L.; Vilain, Eric; Arnadottir, Gudny A.; Sulem, Patrick; Sulem, Telma S.; Stefansson, Kari; Massey, Shavonne; Ginn, Natalie; Poduri, Annapurna; D'Gama, Alissa M.; Valentine, Rozalia; Trowbridge, Sara K.; Murali, Chaya N.; Franciskovich, Rachel; Tran, Yen; Webb, Bryn D.; Keppler-Noreuil, Kim M.; Hall, April L.; Mcgivern, Bobbi; Monaghan, Kristin G.; Sacoto, Maria J. Guillen; Baldridge, Dustin; Silverman, Gary A.; Dahiya, Sonika; Turner, Tychele N.; Schedl, Tim; Corbin, Joshua G.; Pak, Stephen C.; Zohn, Irene E.; Gurnett, Christina A. Share Save Share Save Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring programGiugliani, Roberto; Gonzalez-Meneses, Antonio; Scarpa, Maurizio; Burton, Barbara; Wang, Raymond; Martins, Esmeralda; Oussoren, Esmeralda; Hennermann, Julia B.; Chabrol, Brigitte; Grant, Christina L.; Sun, Angela; Durand, Consuelo; Hetzer, Joel; Malkus, Betsy; Marsden, Deborah; Merritt II, J. Lawrence Share Save Early clinical response and continued safety with vestronidase ALFA for the treatment of mucopolysaccharidosis VII (MPS VII)Burton, Barbara; Giugliani, Roberto; Gonzalez-Meneses, Antonio; Grant, Christina Lee; Scarpa, Maurizio; Sun, Angela; Wang, Raymond; Van der Ploeg, Ans; Martins, Esmeralda; Durand, Consuelo; Chabrol, Brigitte; Hetzer, Joel; Malkus, Betsy; Cohen, Jeanne; Marsden, Deborah; Merritt, J. Lawrence, II Share Save Share Save The clinical challenge of new variants encountered in newborn screening: The example of ASN290ASP in GAATortorelli, Silvia; Smith, Wendy; Pillai, Nishitha; Grant, Christina Lee; Kuperman, KeriAnn; Fabie, Noelle; Wright, Erica; Sapp, Katie; Baker, Craig; Keller, Alyssa; Peck, Dawn Share Save Death associated with brain herniation in classical MSUD patients from underrepresented populationsLeon, Eyby; Shur, Natasha; Chapman, Kimberly; Grant, Christina Lee; Fraser, Jamie; Regier, Debra; Vergano, Samantha; MacLeod, Erin; Starin, Danielle; Harmon, Jennifer; Laso, Maria Cristina Share Save Share Save Share Save Share Save Share Save Share Save Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)Malkus, Betsy C.; Cohen, Jeanne M.; Grant, Christina L.; Scarpa, Maurizio; Sun, Angela; Wang, Raymond Y.; Burton, Barbara K.; Oussoren, Esmeralda; Martins, Esmeralda E.; Durand, Consuelo; Chabrol, Brigitte; Ortiz, Lionel; Hetzer, Joel; Marsden, Deborah; Merritt, J. Lawrence, II; Giugliani, Roberto; Gonzalez-Meneses, Antonio Share Save Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multi-center disease monitoring program (DMP)Giugliani, Roberto; Grant, Christina L.; Scarpa, Maurizio; Sun, Angela; Wang, Raymond Y.; Burton, Barbara K.; Oussoren, Esmeralda; Durand, Consuelo; Martins, Esmeralda E.; Chabrol, Brigitte; Hetzer, Joel; Marsden, Deborah; Merritt, J. Lawrence, II; Gonzalez-Meneses, Antonio Share Save Assessing the feasibility of an intervention for adolescents and parents transitioning out of paediatric eating disorder services: A mixed methods studyNicula, Maria; Kimber, Melissa; Boylan, Khrista; Grant, Christina; Laliberte, Michele; Miller, Kathryn; Dimitropoulos, Gina; Trollope, Karen; Webb, Cheryl; Couturier, Jennifer Share Save 
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