Showing 1 - 13 results of 13 for search 'Paul Harmatz', query time: 0.98s
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Current and new therapies for mucopolysaccharidoses by Monica Penon-Portmann, David R. Blair, Paul Harmatz
Published 2023-02-01
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P027: Analysis of glycosaminoglyans in biological fluids reveals diverging trends in heparan sulfate, dermatan sulfate and chondroitine sulfate concentrations with age* by Iskren Menkovic, James Beasley, Haoyue Zhang, Billie Lianoglou, Jingwei Yu, Ashlee Stiles, Paul Harmatz, Sarah Young
Published 2023-01-01
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P194: Persistence of growth promoting effects in children with achondroplasia over seven years: Update from phase II extension study with vosoritide* by Julie Hoover-Fong, Melita Irving, Carlos Bacino, Joel Charrow, Valérie Cormier-Daire, Lynda Polgreen, Paul Harmatz, Alice Huntsman-Labed, Elena Fisheleva, Ian Sabir, Jonathan Day, John Phillips, Ravi Savarirayan
Published 2023-01-01
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P144: Persistence of growth-promoting effects in children with achondroplasia up to 7 years: Update from phase 2 extension study with vosoritide* by Julie Hoover-Fong, Melita Irving, Carlos Bacino, Joel Charrow, Carlos Prada, Valerie Cormier-Daire, Lynda Polgreen, Paul Harmatz, Sajda Ghani, Elena Fisheleva, Andrea Low, Jonathan Day, John Phillips, III, Ravi Savarirayan
Published 2024-01-01
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P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation by Paul Harmatz, Robert Wynn, Ashish Gupta, Sandhya Kharbanda, Caroline Lindemans, Rebecca Ahrens-Nicklas, Peter van Hasselt, Troy Lund, Timothy Olson, Francesca Tucci, Leonie Martin, Nathalie Boeglin, Jean Brooks, Su Syonmez, Laura Campbell, Simon Jones, Paul Orchard, Maria Ester Bernardo
Published 2024-01-01
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P131: Persistence of growth-promoting effects in infants and toddlers with achondroplasia: Results from a phase II extension study with vosoritide* by Ravi Savarirayan, William Wilcox, Paul Harmatz, John Phillips, III, Lynda Polgreen, Louise Tofts, Keiichi Ozono, Paul Arundel, Melita Irving, Carlos Bacino, Donald Basel, Ricki Carroll, Joel Charrow, Hiroshi Mochizuki, Yumiko Kotani, Howard Saal, Lingling Han, Andrea Low, Elena Fisheleva, Jonathan Day
Published 2024-01-01
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O22: A randomized controlled trial of vosoritide in infants and toddlers with achondroplasia* by Carlos Bacino, Ravi Savarirayan, William Wilcox, Paul Harmatz, John Phillips, Lynda Polgreen, Louise Tofts, Keiichi Ozono, Paul Arundel, Melita Irving, Donald Basel, Michael Bober, Joel Charrow, Hiroshi Mochizuki, Yumiko Kotani, Howard Saal, George Jeha, Lynn Han, Elena Fisheleva, Alice Huntsman-Labed, Jonathan Day
Published 2023-01-01
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A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B by Nicole Muschol, Anja Koehn, Katharina von Cossel, Ilyas Okur, Fatih Ezgu, Paul Harmatz, Maria J. de Castro Lopez, Maria Luz Couce, Shuan-Pei Lin, Spyros Batzios, Maureen Cleary, Martha Solano, Igor Nestrasil, Brian Kaufman, Adam J. Shaywitz, Stephen M. Maricich, Bernice Kuca, Joseph Kovalchin, Eric Zanelli
Published 2023-01-01
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P193: Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: Update from phase 3 extension study* by Julie Hoover-Fong, Ravi Savarirayan, Louise Tofts, Melita Irving, William Wilcox, Carlos Bacino, Rosendo Ullot Font, Paul Harmatz, Frank Rutsch, Michael Bober, Lynda Polgreen, Ignacio Ginebreda, Klaus Mohnike, Joel Charrow, Daniel Hoernschemeyer, Keiichi Ozono, Yasemin Alanay, Paul Arundel, Shoji Kagami, Natsuo Yasui, Klane White, Howard Saal, Antonio Leiva-Gea, Felipe Luna-González, Hiroshi Mochizuki, Donald Basel, Dania Porco, Kala Jayaram, Elena Fisheleva, Sue Lawrinson, Jonathan Day
Published 2023-01-01
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P141: Persistent growth-promoting effects of vosoritide in children with achondroplasia is accompanied by improvement in physical aspects of quality of life* by Ravi Savarirayan, Louise Tofts, Melita Irving, William Wilcox, Carlos Bacino, Julie Hoover-Fong, Rosendo Ullot Font, Paul Harmatz, Frank Rutsch, Ricki Carroll, Lynda Polgreen, Ignacio Ginebreda, Klaus Mohnike, Joel Charrow, Carlos Prada, Daniel Hoernschemeyer, Keiichi Ozono, Takuo Kubota, Yasemin Alanay, Paul Arundel, Yumiko Kotani, Natsuo Yasui, Klane White, Shelley Brandstetter, Howard Saal, Antonio Leiva-Gea, Felipe Luna-González, Hiroshi Mochizuki, Asako Tajima, Donald Basel, Elena Fisheleva, Richard Rowell, Alice Huntsman Labed, Jonathan Day
Published 2024-01-01
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P139: Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 4 years: Update from phase 3 extension study* by Ravi Savarirayan, Louise Tofts, Melita Irving, William Wilcox, Carlos Bacino, Julie Hoover-Fong, Rosendo Ullot Font, Paul Harmatz, Frank Rutsch, Ricki Carroll, Lynda Polgreen, Ignacio Ginebreda, Klaus Mohnike, Joel Charrow, Carlos Prada, Daniel Hoernschemeyer, Keiichi Ozono, Takuo Kubota, Yasemin Alanay, Paul Arundel, Yumiko Kotani, Natsuo Yasui, Klane White, Shelley Brandstetter, Howard Saal, Antonio Leiva-Gea, Felipe Luna-González, Hiroshi Mochizuki, Asako Tajima, Donald Basel, Elena Fisheleva, Andrea Low, Sue Lawrinson, Jonathan Day
Published 2024-01-01
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