Urine phosphoethanolamine is a specific biomarker for hypophosphatasia in adults von Shajani-Yi, Zahra, Ayala-Lopez, Nadia, Black, Margo, Dahir, Kathryn McCrystal

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Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia von Dahir, Kathryn M, Shannon, Amy, Dunn, Derek, Voegtli, Walter, Dong, Qunming, Hasan, Jawad, Pradhan, Rajendra, Pelto, Ryan, Pan, Wei-Jian

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Clinical profiles of treated and untreated adults with hypophosphatasia in the Global HPP Registry von Dahir, Kathryn M, Seefried, Lothar, Kishnani, Priya S, Petryk, Anna, Högler, Wolfgang, Linglart, Agnès, Martos-Moreno, Gabriel Ãngel, Ozono, Keiichi, Fang, Shona, Rockman-Greenberg, Cheryl

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Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry von Kishnani, Priya S, Martos-Moreno, Gabriel Ángel, Linglart, Agnès, Petryk, Anna, Messali, Andrew, Fang, Shona, Rockman-Greenberg, Cheryl, Ozono, Keiichi, Högler, Wolfgang, Seefried, Lothar, Dahir, Kathryn M

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Pharmacodynamics of asfotase alfa in adults with pediatric-onset hypophosphatasia von Seefried, Lothar, Kishnani, Priya S., Moseley, Scott, Denker, Andrew E., Watsky, Eric, Whyte, Michael P., Dahir, Kathryn M.

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Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a post hoc analysis of the randomized, double-blind, placebo-controlled... von Keen, Richard, Dahir, Kathryn M, McGinniss, Jennifer, Sanchez, Robert J, Mellis, Scott, Economides, Aris N, Di Rocco, Maja, Orcel, Philippe, Roux, Christian, Tabarkiewicz, Jacek, Bachiller-Corral, Javier, Cheung, Angela M, Al Mukaddam, Mona, Mohammadi, Kusha, Gu, Jing, Srinivasan, Dushyanth, Trotter, Dinko Gonzalez, Eekhoff, E Marelise W, Kaplan, Frederick S, Pignolo, Robert J

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Setrusumab for the treatment of osteogenesis imperfecta: 12-month results from the phase 2b asteroid study von Glorieux, Francis H, Langdahl, Bente, Chapurlat, Roland, De Beur, Suzanne Jan, Sutton, Vernon Reid, Poole, Kenneth E S, Dahir, Kathryn M, Orwoll, Eric S, Willie, Bettina M, Mikolajewicz, Nicholas, Zimmermann, Elizabeth, Hosseinitabatabaei, Seyedmahdi, Ominsky, Michael S, Saville, Chris, Clancy, James, MacKinnon, Alastair, Mistry, Arun, Javaid, Muhammad K

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Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial von Di Rocco, Maja, Forleo-Neto, Eduardo, Pignolo, Robert J., Keen, Richard, Orcel, Philippe, Funck-Brentano, Thomas, Roux, Christian, Kolta, Sami, Madeo, Annalisa, Bubbear, Judith S., Tabarkiewicz, Jacek, Szczepanek, Małgorzata, Bachiller-Corral, Javier, Cheung, Angela M., Dahir, Kathryn M., Botman, Esmée, Raijmakers, Pieter G., Al Mukaddam, Mona, Tile, Lianne, Portal-Celhay, Cynthia, Sarkar, Neena, Hou, Peijie, Musser, Bret J., Boyapati, Anita, Mohammadi, Kusha, Mellis, Scott J., Rankin, Andrew J., Economides, Aris N., Trotter, Dinko Gonzalez, Herman, Gary A., O’Meara, Sarah J., DelGizzi, Richard, Weinreich, David M., Yancopoulos, George D., Eekhoff, E. Marelise W., Kaplan, Frederick S.

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Hypophosphatasia diagnosis: current state of the art and proposed diagnostic criteria for children and adults von Khan, Aliya A., Brandi, Maria Luisa, Rush, Eric T., Ali, Dalal S., Al-Alwani, Hatim, Almonaei, Khulod, Alsarraf, Farah, Bacrot, Severine, Dahir, Kathryn M., Dandurand, Karel, Deal, Chad, Ferrari, Serge Livio, Giusti, Francesca, Guyatt, Gordon, Hatcher, Erin, Ing, Steven W., Javaid, Muhammad Kassim, Khan, Sarah, Kocijan, Roland, Linglart, Agnes, M’Hiri, Iman, Marini, Francesca, Nunes, Mark E., Rockman-Greenberg, Cheryl, Roux, Christian, Seefried, Lothar, Simmons, Jill H., Starling, Susan R., Ward, Leanne M., Yao, Liang, Brignardello-Petersen, Romina, Lewiecki, E. Michael

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Clinical Profiles of Children with Hypophosphatasia Prior to Treatment with Enzyme Replacement Therapy: An Observational Analysis from the Global HPP Registry von Martos-Moreno, Gabriel Ángel, Rockman-Greenberg, Cheryl, Ozono, Keiichi, Petryk, Anna, Kishnani, Priya S, Dahir, Kathryn M, Seefried, Lothar, Fang, Shona, Högler, Wolfgang, Linglart, Agnès

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An update on clinical presentation and responses to therapy of patients with hereditary hypophosphatemic rickets with hypercalciuria (HHRH) von Zhu, Zewu, Bo-Ran Ho, Bryan, Chen, Alyssa, Amrhein, James, Apetrei, Andreea, Carpenter, Thomas Oliver, Lazaretti-Castro, Marise, Colazo, Juan Manuel, McCrystal Dahir, Kathryn, Geßner, Michaela, Gurevich, Evgenia, Heier, Cathrine Alsaker, Simmons, Jill Hickman, Hunley, Tracy Earl, Hoppe, Bernd, Jacobsen, Christina, Kouri, Anne, Ma, Nina, Majumdar, Sachin, Molin, Arnaud, Nokoff, Natalie, Ott, Susan M., Peña, Helena Gil, Santos, Fernando, Tebben, Peter, Topor, Lisa Swartz, Deng, Yanhong, Bergwitz, Clemens

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Impact of muscular symptoms and/or pain on disease characteristics, disability, and quality of life in adult patients with hypophosphatasia: A cross-sectional analysis from the Glo... von Dahir, Kathryn M, Kishnani, Priya S, Martos-Moreno, Gabriel Ángel, Linglart, Agnès, Petryk, Anna, Rockman-Greenberg, Cheryl, Martel, Samantha E, Ozono, Keiichi, Högler, Wolfgang, Seefried, Lothar

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New insights into the landscape of ALPL gene variants in patients with hypophosphatasia from the Global HPP Registry von Kishnani, Priya S., Seefried, Lothar, Dahir, Kathryn M., Martos‐Moreno, Gabriel Ángel, Linglart, Agnès, Petryk, Anna, Mowrey, William R., Fang, Shona, Ozono, Keiichi, Högler, Wolfgang, Rockman‐Greenberg, Cheryl

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The challenge of hypophosphatasia diagnosis in adults: results from the HPP International Working Group Literature Surveillance von Brandi, Maria Luisa, Khan, Aliya A., Rush, Eric T., Ali, Dalal S., Al-Alwani, Hatim, Almonaei, Khulod, Alsarraf, Farah, Bacrot, Severine, Dahir, Kathryn M., Dandurand, Karel, Deal, Chad, Ferrari, Serge Livio, Giusti, Francesca, Guyatt, Gordon, Hatcher, Erin, Ing, Steven W., Javaid, Muhammad Kassim, Khan, Sarah, Kocijan, Roland, Lewiecki, E. Michael, Linglart, Agnes, M’Hiri, Iman, Marini, Francesca, Nunes, Mark E., Rockman-Greenberg, Cheryl, Seefried, Lothar, Simmons, Jill H., Starling, Susan R., Ward, Leanne M., Yao, Liang, Brignardello-Petersen, Romina, Roux, Christian

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X-Linked Hypophosphatemia: A New Era in Management von Dahir, Kathryn, Roberts, Mary Scott, Krolczyk, Stan, Simmons, Jill H

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Proposed diagnostic criteria for the diagnosis of hypophosphatasia in children and adolescents: results from the HPP International Working Group von Rush, Eric, Brandi, Maria Luisa, Khan, Aliya, Ali, Dalal S., Al-Alwani, Hatim, Almonaei, Khulod, Alsarraf, Farah, Bacrot, Severine, Dahir, Kathryn M., Dandurand, Karel, Deal, Chad, Ferrari, Serge Livio, Giusti, Francesca, Guyatt, Gordon, Hatcher, Erin, Ing, Steven W., Javaid, Muhammad Kassim, Khan, Sarah, Kocijan, Roland, Lewiecki, E. Michael, Linglart, Agnes, M’Hiri, Iman, Marini, Francesca, Nunes, Mark E., Rockman-Greenberg, Cheryl, Roux, Christian, Seefried, Lothar, Starling, Susan R., Ward, Leanne, Yao, Liang, Brignardello-Petersen, Romina, Simmons, Jill H.

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Correction: Hypophosphatasia diagnosis: current state of the art and proposed diagnostic criteria for children and adults von Khan, Aliya A., Brandi, Maria Luisa, Rush, Eric T., Ali, Dalal S., Al-Alwani, Hatim, Almonaei, Khulod, Alsarraf, Farah, Bacrot, Severine, Dahir, Kathryn M., Dandurand, Karel, Deal, Chad, Ferrari, Serge Livio, Giusti, Francesca, Guyatt, Gordon, Hatcher, Erin, Ing, Steven W., Javaid, Muhammad Kassim, Khan, Sarah, Kocijan, Roland, Linglart, Agnes, M’Hiri, Iman, Marini, Francesca, Nunes, Mark E., Rockman-Greenberg, Cheryl, Roux, Christian, Seefried, Lothar, Simmons, Jill H., Starling, Susan R., Ward, Leanne M., Yao, Liang, Brignardello-Petersen, Romina, Michael Lewiecki, E.

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Rare Causes of Musculoskeletal Pain: Thinking beyond Common Rheumatologic Diseases von Charles, Julia F., Malabanan, Alan O., Krolczyk, Stan, Dahir, Kathryn M.

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Real-World Clinical and Healthcare Resource Burden Among Burosumab-Naïve Patients With Familial Hypophosphatemia von Imel, Erik A, Li, Zhiyi, Heerssen, Heather M, Princic, Nicole, Schwartz, Hana, Zhao, Yang, Dahir, Kathryn M

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Improvement in quality of life after asfotase alfa treatment in adults with pediatric-onset hypophosphatasia: data from 5 patient-reported outcome measures von Dahir, Kathryn M, Ing, Steven W, Deal, Chad, Messali, Andrew, Bates, Toby, Rush, Eric T

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