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8 results on '"Jayaram, Kala"'

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

Author

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, and Day, Jonathan RS

Subjects
Clinical Trials and Supportive Activities, Clinical Research, Pediatric, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Decent Work and Economic Growth, Achondroplasia, Child, Double-Blind Method, Humans, Natriuretic Peptide, C-Type, Treatment Outcome, Genetics, Clinical Sciences, Genetics & Heredity
Abstract

PurposeAchondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported.MethodsAfter completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.ResultsIn children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104. In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104. No new adverse effects of vosoritide were detected.ConclusionVosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years.

Published
2021

4. Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Author

Mahlangu, Johnny, Kaczmarek, Radoslaw, von Drygalski, Annette, Shapiro, Susan, Chou, Sheng-Chieh, Ozelo, Margareth C, Kenet, Gili, Peyvandi, Flora, Wang, Michael, Madan, Bella, Key, Nigel S, Laffan, Michael, Dunn, Amy L, Mason, Jane, Quon, Doris V, Symington, Emily, Leavitt, Andrew D, Oldenburg, Johannes, Chambost, Hervé, Reding, Mark T, Jayaram, Kala, Yu, Hua, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B, Henshaw, Joshua, Robinson, Tara M, Wong, Wing Yen, Pipe, Steven W, GENEr8-1 Trial Group, Hermans, Cédric, UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, and UCL - (SLuc) Centre de malformations vasculaires congénitales

Subjects
Male, Factor VIII, Recombinant Fusion Proteins, Gene Transfer Techniques, Humans, Hemorrhage, General Medicine, Hemophilia A, Half-Life
Abstract

BACKGROUND: Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a phase 3 study of the efficacy and safety of valoctocogene roxaparvovec therapy evaluated after 52 weeks in men with severe hemophilia A have been published previously. METHODS: We conducted an open-label, single-group, multicenter, phase 3 trial in which 134 men with severe hemophilia A who were receiving factor VIII prophylaxis received a single infusion of 6×1013 vector genomes of valoctocogene roxaparvovec per kilogram of body weight. The primary end point was the change from baseline in the annualized rate of treated bleeding events at week 104 after receipt of the infusion. The pharmacokinetics of valoctocogene roxaparvovec were modeled to estimate the bleeding risk relative to the activity of transgene-derived factor VIII. RESULTS: At week 104, a total of 132 participants, including 112 with data that were prospectively collected at baseline, remained in the study. The mean annualized treated bleeding rate decreased by 84.5% from baseline (P

Published
2023

6. P193: Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: Update from phase 3 extension study*

Author

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

Published
2023
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7. Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Author

Ozelo, Margareth C., primary, Mahlangu, Johnny, additional, Pasi, K. John, additional, Giermasz, Adam, additional, Leavitt, Andrew D., additional, Laffan, Michael, additional, Symington, Emily, additional, Quon, Doris V., additional, Wang, Jiaan-Der, additional, Peerlinck, Kathelijne, additional, Pipe, Steven W., additional, Madan, Bella, additional, Key, Nigel S., additional, Pierce, Glenn F., additional, O’Mahony, Brian, additional, Kaczmarek, Radoslaw, additional, Henshaw, Joshua, additional, Lawal, Adebayo, additional, Jayaram, Kala, additional, Huang, Mei, additional, Yang, Xinqun, additional, Wong, Wing Y., additional, and Kim, Benjamin, additional

Published
2022
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8. Vosoritide treatment accelerates bone growth in children with achondroplasia

Author

Savarirayan, Ravi, primary, Irving, Melita, additional, Hoover-Fong, Julie, additional, Bacino, Carlos A, additional, Ozono, Keiichi, additional, Mohnike, Klaus, additional, Cormier-Daire, Valerie, additional, Leiva-Gea, Antonio, additional, Alanay, Yasemin, additional, Andrews, Mary, additional, Crews, Chandler, additional, Klafehn, Cristina, additional, Jayaram, Kala, additional, Jeha, George S, additional, Fisheleva, Elena, additional, Huntsman-Labed, Alice, additional, and Day, Jonathan, additional

Published
2021
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