A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease

por Byrne, Barry J., Geberhiwot, Tarekegn, Barshop, Bruce A., Barohn, Richard, Hughes, Derralynn, Bratkovic, Drago, Desnuelle, Claude, Laforet, Pascal, Mengel, Eugen, Roberts, Mark, Haroldsen, Peter, Reilley, Kristin, Jayaram, Kala, Yang, Ke, Walsh, Liron
Publicado 2017

Pharmacokinetics and Exposure–Response of Vosoritide in Children with Achondroplasia

por Chan, Ming Liang, Qi, Yulan, Larimore, Kevin, Cherukuri, Anu, Seid, Lori, Jayaram, Kala, Jeha, George, Fisheleva, Elena, Day, Jonathan, Huntsman-Labed, Alice, Savarirayan, Ravi, Irving, Melita, Bacino, Carlos A., Hoover-Fong, Julie, Ozono, Keiichi, Mohnike, Klaus, Wilcox, William R., Horton, William A., Henshaw, Joshua
Publicado 2021

Persistent and Stable Growth Promoting Effects of Vosoritide in Children With Achondroplasia for up to 2 Years: Results From the Ongoing Phase 3 Extension Study

por Savarirayan, Ravi, Tofts, Louise, Irving, Melita, Wilcox, William, Bacino, Carlos A, Hoover-Fong, Julie, Font, Rosendo Ullot, Harmatz, Paul, Rutsch, Frank, Bober, Michael B, Polgreen, Lynda, Ginebreda, Ignacio, Mohnike, Klaus, Charrow, Joel, Hoernschemeyer, Daniel, Ozono, Keiichi, Alanay, Yasemin, Arundel, Paul, Kagami, Shoji, Yasui, Natsuo, White, Klane, Saal, Howard, Leiva-Gea, Antonio, Luna-González, Felipe, Mochizuki, Hiroshi, Basel, Donald, Porco, Dania, Jayaram, Kala, Fisheleva, Elena, Han, Lynn, Day, Jonathan
Publicado 2021

SAT-LB18 A Randomized Controlled Trial of Vosoritide in Children With Achondroplasia

por Savarirayan, Ravi, Tofts, Louise, Irving, Melita, Wilcox, William, Bacino, Carlos A, Hoover-Fong, Julie, Font, Rosendo Ullot, Harmatz, Paul, Rutsch, Frank, Bober, Michael B, Polgreen, Lynda E, Ginebreda, Ignacio, Mohnike, Klaus, Charrow, Joel, Hoernschmeyer, Daniel, Ozono, Keiichi, Alanay, Yasemin, Arundel, Paul, Kagami, Shoji, Yasui, Natsuo, White, Klane, Saal, Howard M, Leiva-Gea, Antonio, Luna-Gonzáles, Felipe, Mochizuki, Hiroshi, Basel, Donald, Porco, Dania M, Jayaram, Kala, Fisheleva, Elena, Huntsman-Labed, Alice, Day, Jonathan
Publicado 2020

Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study

por Savarirayan, Ravi, Tofts, Louise, Irving, Melita, Wilcox, William R., Bacino, Carlos A., Hoover-Fong, Julie, Font, Rosendo Ullot, Harmatz, Paul, Rutsch, Frank, Bober, Michael B., Polgreen, Lynda E., Ginebreda, Ignacio, Mohnike, Klaus, Charrow, Joel, Hoernschemeyer, Daniel, Ozono, Keiichi, Alanay, Yasemin, Arundel, Paul, Kotani, Yumiko, Yasui, Natsuo, White, Klane K., Saal, Howard M., Leiva-Gea, Antonio, Luna-González, Felipe, Mochizuki, Hiroshi, Basel, Donald, Porco, Dania M., Jayaram, Kala, Fisheleva, Elena, Huntsman-Labed, Alice, Day, Jonathan R. S.
Publicado 2021