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Fractures are highly correlated with bone density and inversely correlated with bone turnover markers in autosomal dominant osteopetrosis Econs, Michael J.; Warden, Stuart J.; Liu, Ziyue; Niziolek, Paul J.; Parks-Schenck, Corinne; Gebregziabher, Netsanet; Gerard-O'Riley, Rita L.; Hart, Marian; Polgreen, Lynda E.; Imel, Erik A. 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
Anakinra in Sanfilippo syndrome: a phase 1/2 trial (Jul, 10.1038/s41591-024-03079-3, 2024) Polgreen, Lynda E.; Chen, Agnes H.; Pak, Youngju; Luzzi, Anna; Garval, Adolfo Morales; Acevedo, Jonathan; Bitan, Gal; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B. Share Save
Anakinra in Sanfilippo syndrome: a phase 1/2 trial Polgreen, Lynda E.; Chen, Agnes H.; Pak, Youngju; Luzzi, Anna; Garval, Adolfo Morales; Acevedo, Jonathan; Bitan, Gal; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B. Share Save
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New blood-based biomarkers for Sanfilippo syndrome Bitan, Gal; Taha, Hash Brown; Siddique, Ibrar; Chamoun, Lea M.; Mohseni, Kimia; Morris, Alanna J.; Luzzi, Anna; Acevedo, Jonathan; Morales, Adolfo; de la Garza, Giselle Perez; Nour, Elabed; Khijniak, Marie; Fenwick, Leony; Chen, Agnes; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B.; Polgreen, Lynda Share Save
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Impact of anakinra on disordered movement in Sanfilippo syndrome Acevedo, Jonathan; Chen, Agnes; Youngju, Pak; Luzzi, Anna; Morales, Adolfo; Bitan, Gal; Iacovino, Michelina; O'Neill, Cara; Eisengart, Julie B.; Polgreen, Lynda Share Save
Autosomal Dominant Osteopetrosis (ADO) Caused by a Missense Variant in the TCIRG1 Gene Jodeh, Wade; Katz, Amy J.; Hart, Marian; Warden, Stuart J.; Niziolek, Paul; Alam, Imranul; Ing, Steven; Polgreen, Lynda E.; Imel, Erik A.; Econs, Michael J. Share Save
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial Savarirayan, Ravi; Wilcox, William R.; Harmatz, Paul; Phillips, John; Polgreen, Lynda E.; Tofts, Louise; Ozono, Keiichi; Arundel, Paul; Irving, Melita; Bacino, Carlos A.; Basel, Donald; Bober, Michael B.; Charrow, Joel; Mochizuki, Hiroshi; Kotani, Yumiko; Saal, Howard M.; Army, Clare; Jeha, George; Qi, Yulan; Han, Lynn; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan Share Save
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