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• Publication

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Keziah Cook, Shaun P. Forbes, Kelly Adamski, Janice J. Ma, Anita Chawla & Louis P. Garrison Jr

• Publication

Psychometric Validation of the Haemo-QOL-A in Participants with Hemophilia A Treated with Gene Therapy

Jennifer Quinn, Kathleen A. Delaney, Wing Yen Wong, Wolfgang Miesbach, Monika Bullinger

• Publication

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia

Glenn F. Pierce, Sylvia Fong, Brian R. Long, Radoslaw Kaczmarek

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The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects

Paul Harmatz, Karl Eugen Mengel, Roberto Giugliani, Vassili Valayannopoulos, Shuan-Pei Lin, Rossella Parini, Nathalie Guffon, Barbara K. Burton, Christian J. Hendriksz, John Mitchell, Ana Martins, Simon Jones, Norberto Guelbert, Ashok Vellodi, Carla Hollak, Peter Slasor, Celeste Decker

• Publication

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome

Paul R. Harmatz, Karl Eugen Mengel, Roberto Giugliani, Vassili Valayannopoulos, Shuan-Pei Lin, Rossella Parini, Nathalie Guffon, Barbara K. Burton, Christian J. Hendriksz, John J. Mitchell , Ana Maria Martins , Simon A. Jones, Norberto Guelbert, Ashok Vellodi, Frits A. Wijburg, Ke Yang , Peter Slasor, Celeste Decker

• Publication

Diagnosing mucopolysaccharidosis IVA

Timothy C. Wood, Katie Harvey, Michael Beck, Maira Graeff Burin, Yin-Hsiu Chien, Heather J. Church, Vânia D’Almeida, Otto P. van Diggelen, Michael Fietz, Roberto Giugliani, Paul Harmatz, Sara M. Hawley, Wuh-Liang Hwu, David Ketteridge, Zoltan Lukacs, Nicole Miller, Marzia Pasquali, Andrea Schenone, Jerry N. Thompson, Karen Tylee, Chunli Yu, Christian J. Hendriksz

• Publication

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Christian J. Hendriksz, Barbara Burton, Thomas R. Fleming, Paul Harmatz, Derralynn Hughes, Simon A. Jones, Shuan-Pei Lin, Eugen Mengel, Maurizio Scarpa, Vassili Valayannopoulos, Roberto Giugliani, STRIVE Investigators, Peter Slasor, Debra Lounsbury, Wolfgang Dummer

• Publication

Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Christian J. Hendriksz, Rossella Parini, Moeenaldeen D. AlSayed, Julian Raiman, Roberto Giugliani, Martha L. Solano Villarreal, John J. Mitchell, Barbara K. Burton, Norberto Guelbert, Fiona Stewart, Derralynn A. Hughes, Kenneth I. Berger, Peter Slasor m, Robert Matousek, Elaina Jurecki, Adam J. Shaywitz, Paul R. Harmatz

• Publication

Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial

Christian J. Hendriksz, Roberto Giugliani, Paul Harmatz, Eugen Mengel, Nathalie Guffon, Vassili Valayannopoulos, Rossella Parini, Derralynn Hughes, Gregory M. Pastores, Heather A. Lau, Moeenaldeen D. Al-Sayed, Julian Raiman, STRIVE Investigators, Ke Yang, Matthew Mealiffe, Christine Haller

• Publication

Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England

Maureen Cleary, James Davison, Rachel Gould, Tarekegn Geberhiwot, Derralynn Hughes, Jean Mercer, Alexandra Morrison, Elaine Murphy, Saikat Santra, James Jerrett, Swati Mukherjee & Karolina M. Stepien

• Publication

Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa

John J. Mitchell, Barbara K. Burton, Michael B. Bober, Philippe M. Campeau, Shelda Cohen, Sara Dosenovic, Carolyn Ellaway, Kaustuv Bhattacharya, Nathalie Guffon, David Hinds, Alice Lail, Shuan-Pei Lin, Martin Magner, Julian Raiman, Liat Schwartz-Sagi, Karolina M. Stepien

• Publication

Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance

Mehmet Umut Akyol1, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz , Maurizio Scarpa, MPS Consensus Programme Steering Committee and MPS Consensus Programme Co-Chairs

• Publication

Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI

Paul R. Harmatz, Paula Garcia, Nathalie Guffon, Linda M. Randolph, Renée Shediac, Elizabeth Braunlin, Ralph S. Lachman, Celeste Decker

• Publication

The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)

P. Harmatz, C.J. Hendriksz, C. Lampe , J.J. McGill, R. Parini, E. Leão-Teles, V. Valayannopoulos , T.J. Cole , R. Matousek, S. Graham , N. Guffon, A. Quartel, MPS VI Study Group

• Publication

Long-Term Galsulfase Treatment Associated With Improved Survival of Patients With Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-Year Follow-Up From the Survey Study

Adrian Quartel, MD, Paul R. Harmatz, MD , Christina Lampe, MD, Nathalie Guffon, MD, David Ketteridge, MD, Elisa Lea˜o-Teles, MD, Simon A. Jones, MD, and Roberto Giugliani, MD, PhD

• Publication

Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Paul R. Harmatz, Christina Lampe, Rossella Parkini, Reena Sharma, Elisa L. Teles, Julie Johnson, Debbie Sivam, Zlatko Sisic

• Publication

Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Christina Lampea, Paul R. Harmatz, Rossella Parinic, Reena Sharmae, Elisa Leão Telesf, Julie Johnsong, Debbie Sivamg, Zlatko Sisic

• Publication

Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy

Paula Garcia, Dawn Phillips, JoAnn Johnson, Kenneth Martin, Linda M. Randolph, Howard Rosenfeld, Paul Harmatz

• Publication

Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study

Dafne D.G. Horovitz, Emília K.E.A. Leão, Erlane M. Ribeiro, Ana M. Martins, Anneliese L. Barth, João I.C.F. Neri, Marcelo Kerstenetzky, Ana C.M. Siqueira, Bethania F.R. Ribeiro, Chong A. Kim, Francisca C. Santos, José F.S. Franco, Leniza C.L. Lichtvan, Liane R. Giuliani, Maria Do C.S. Rodrigues, Renata C.F. Bonatti, Thaís B. Teixeira , Alexandra Gonçalves, Charles M. Lourenço, Ane S.S. Pereira, Angelina X. Acosta

• Publication

Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

Mehmet Umut Akyol, Tord D. Alden, Hernan Amartino, Jane Ashworth, Kumar Belani, Kenneth I. Berger, Andrea Borgo, Elizabeth Braunlin, Yoshikatsu Eto, Jeffrey I. Gold, Andrea Jester, Simon A. Jones, Cengiz Karsli, William Mackenzie, Diane Ruschel Marinho, Andrew McFadyen, Jim McGill, John J. Mitchell, Joseph Muenzer, Torayuki Okuyama, Paul J. Orchard, Bob Stevens, Sophie Thomas, Robert Walker, Robert Wynn, Roberto Giugliani, Paul Harmatz, Christian Hendriksz, Maurizio Scarpa, MPS Consensus Programme Steering Committee and MPS Consensus Programme Co-Chairs