THU165 PROPEL, PROPEL 2 And PROPEL OLE Studies Of Infigratinib In Children With Achondroplasia: Design And Status Of 3 Ongoing Trials
Savarirayan, Ravi, De Bergua, Josep Maria, Arundel, Paul, McDevitt, Helen, Cormier-Daire, Valerie, Saraff, Vrinda, Skae, Mars, Delgado, Borja, Leiva-Gea, Antonio, Salcedo, Maria, Salles, Jean-Pierre, Nicolino, Marc P, Rossi, Massimiliano, Kannu, Peter, Bober, Michael B, Phillips, John, Saal, Howard, Harmatz, Paul, Burren, Christine, Candler, Toby, Cho, Terry, Muslimova, Elena, Weng, Richard, Rogoff, Daniela, Hoover-Fong, Julie, Irving, Melita
Published in Journal of the Endocrine Society (05.10.2023)
Published in Journal of the Endocrine Society (05.10.2023)
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Journal Article
OR27-03 Oral Infigratinib Treatment Is Well Tolerated And Significantly Increases Height Velocity In Children With Achondroplasia: Month 6 Results From The PROPEL 2 Dose-finding Study
Savarirayan, Ravi, De Bergua, Josep Maria, Arundel, Paul, Salles, Jean Pierre, Saraff, Vrinda, Delgado, Borja, Leiva-Gea, Antonio, McDevitt, Helen, Nicolino, Marc P, Rossi, Massimiliano, Salcedo, Maria, Cormier-Daire, Valerie, Skae, Mars, Kannu, Peter, Bober, Michael B, Phillips, John, Saal, Howard, Harmatz, Paul, Burren, Christine Pamela, Candler, Toby, Cho, Terry, Muslimova, Elena, Weng, Richard, Raj, Supriya, Hoover-Fong, Julie, Irving, Melita, Rogoff, Daniela
Published in Journal of the Endocrine Society (05.10.2023)
Published in Journal of the Endocrine Society (05.10.2023)
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Journal Article
THU181 Evaluation Of Bone Mineral Density In A Cohort Of Children With ACH Participating In The PROPEL 2 Study Of Infigratinib
Savarirayan, Ravi, De Bergua, Josep Maria, Arundel, Paul, Salles, Jean Pierre, Leiva-Gea, Antonio, Irving, Melita, Saraff, Vrinda, McDevitt, Helen, Salcedo, Maria, Nicolino, Marc P, Cormier-Daire, Valerie, Kannu, Peter, Skae, Mars, Bober, Michael B, Phillips III, John, Candler, Toby, Harmatz, Paul, Saal, Howard, Hoover-Fong, Julie, Muslimova, Elena, Cho, Terry, Weng, Richard, Rogoff, Daniela
Published in Journal of the Endocrine Society (05.10.2023)
Published in Journal of the Endocrine Society (05.10.2023)
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Journal Article
Chondrodysplasia and growth failure in children after early hematopoietic stem cell transplantation for non‐oncologic disorders
Botto, Lorenzo D., Meeths, Marie, Campos‐Xavier, Belinda, Bergamaschi, Rosalba, Mazzanti, Laura, Scarano, Emanuela, Finocchi, Andrea, Cancrini, Caterina, Zirn, Birgit, Kühnle, Ingrid, Kramm, Christof Maria, Alanay, Yasemin, Jones, Wendy D., Irving, Melita, Sabir, Ataf, Henter, Jan‐Inge, Borgström, Birgit, Nordgren, Ann, Hammarsjö, Anna, Putti, Caterina, Mozzato, Chiara, Zuccarello, Daniela, Nishimura, Gen, Bonafè, Luisa, Grigelioniene, Giedre, Unger, Sheila, Superti‐Furga, Andrea
Published in American journal of medical genetics. Part A (01.02.2021)
Published in American journal of medical genetics. Part A (01.02.2021)
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Journal Article
The CHD4-related syndrome: a comprehensive investigation of theclinical spectrum, genotype–phenotype correlations, and molecular basis
Weiss, Karin, Lazar, Hayley P, Kurolap Alina, Martinez, Ariel F, Paperna Tamar, Cohen, Lior, Smeland, Marie F, Whalen, Sandra, Heide Solveig, Keren, Boris, Terhal Pauline, Irving Melita, Takaku Motoki, Roberts, John D, Petrovich, Robert M, Schrier Vergano Samantha A, Kenney, Amy, Hove Hanne, DeChene, Elizabeth, Quinonez, Shane C, Colin, Estelle, Ziegler, Alban, Rumple Melissa, Jain Mahim, Monteil, Danielle, Roeder, Elizabeth R, Nugent, Kimberly, van Haeringen Arie, Gambello, Michael, Avni, Santani, Medne Līvija, Krock Bryan, Skraban Cara M, Zackai, Elaine H, Dubbs, Holly A, Smol, Thomas, Ghoumid Jamal, Parker, Michael J, Wright, Michael, Turnpenny, Peter, Clayton-Smith, Jill, Metcalfe, Kay, Kurumizaka Hitoshi, Gelb, Bruce D, Baris Feldman Hagit, Campeau, Philippe M, Muenke Maximilian, Wade, Paul A, Lachlan, Katherine
Published in Genetics in medicine (01.02.2020)
Published in Genetics in medicine (01.02.2020)
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Journal Article
The T-Cell Receptor Can Bind to the Peptide-Bound Major Histocompatibility Complex and Uncomplexed β 2 -Microglobulin through Distinct Binding Sites
Merkle, Patrick S, Irving, Melita, Hongjian, Song, Ferber, Mathias, Jørgensen, Thomas J D, Scholten, Kirsten, Luescher, Immanuel, Coukos, George, Zoete, Vincent, Cuendet, Michel A, Michielin, Olivier, Rand, Kasper D
Published in Biochemistry (Easton) (01.08.2017)
Published in Biochemistry (Easton) (01.08.2017)
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Journal Article
입양 전달 요법에서 T 림프구 및 NK 세포의 확장 및 분화를 위한 방법
LEON MONZON KALET, ORTIZ MIRANDA YAQUELIN, IRVING MELITA, CRIBIOLI ELISABETTA, CORRIA OSORIO ANGEL DE JESUS, MONTALVO BEREAU GALIA MAGELA, COUKOS GEORGE
Year of Publication 03.02.2022
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Year of Publication 03.02.2022
Patent
PSAT105 Evaluation of Body Mass Index and Metabolic Parameters in Children with Achondroplasia Participating in the PROPEL Study
Rogoff, Daniela, Bergua, Josep Maria De, Savarirayan, Ravi, Arundel, Paul, Salles, Jean Pierre, Leiva-Gea, Antonio, Irving, Melita, Saraff, Vrinda, McDevitt, Helen, Santos-Simarro, Fernando, Nicolino, Marc, Cormier-Daire, Valerie, Kannu, Peter, Skae, Mars, Bober, Michael B, Phillips III, John, Burren, Christine, Harmatz, Paul, Saal, Howard, Hoover-Fong, Julie, Muslimova, Elena, Cho, Terry, Weng, Richard
Published in Journal of the Endocrine Society (01.11.2022)
Published in Journal of the Endocrine Society (01.11.2022)
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Journal Article
RF26 | PMON326 Medical History of Children Enrolled in PROPEL: A Prospective Clinical Assessment Study in Children with Achondroplasia
Irving, Melita, De Bergua, Josep Maria, Rogoff, Daniela, Savarirayan, Ravi, Arundel, Paul, Pierre Salles, Jean, Leiva-Gea, Antonio, Saraff, Vrinda, McDevitt, Helen, Santos-Simarro, Fernando, Nicolino, Marc, Cormier-Daire, Valerie, Kannu, Peter, Skae, Mars, Bober, Michael B, Phillips III, John, Burren, Christine, Harmatz, Paul, Saal, Howard, Hoover-Fong, Julie, Weng, Richard, Muslimova, Elena, Cho, Terry
Published in Journal of the Endocrine Society (01.11.2022)
Published in Journal of the Endocrine Society (01.11.2022)
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Journal Article
PSAT106 Infigratinib in Children with Achondroplasia: Design of the PROPEL, PROPEL2 and PROPEL OLE Studies
Muslimova, Elena, De Bergua, Josep Maria, Savarirayan, Ravi, Arundel, Paul, McDevitt, Helen, Cormier-Daire, Valerie, Saraff, Vrinda, Skae, Mars, Delgado, Borja, Leiva-Gea, Antonio, Santos-Simarro, Fernando, Pierre Salles, Jean, Nicolino, Marc, Rossi, Massimiliano, Kannu, Peter, Bober, Michael B, Phillips III, John, Saal, Howard, Harmatz, Paul, Burren, Christine, Gotway, Garrett, Cho, Terry, Weng, Richard, Rogoff, Daniela, Hoover-Fong, Julie, Irving, Melita
Published in Journal of the Endocrine Society (01.11.2022)
Published in Journal of the Endocrine Society (01.11.2022)
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Journal Article
LBMON196 A Randomized Controlled Trial Of Vosoritide In Infants And Toddlers With Achondroplasia
Savarirayan, Ravi, Wilcox, William W, Harmatz, Paul, III, John Phillips, Polgreen, Lynda E, Tofts, Louise, Ozono, Keiichi, Arundel, Paul, Irving, Melita, Bacino, Carlos A, Basel, Donald, Bober, Michael B, Charrow, Joel, Mochizuki, Hiroshi, Kotani, Yumiko, Saal, Howard M, Jeha, George, Han, Lynn, Fisheleva, Elena, Huntsman-Labed, Alice, Day, Jonathan
Published in Journal of the Endocrine Society (01.11.2022)
Published in Journal of the Endocrine Society (01.11.2022)
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Journal Article
Preclinical Evaluation and Dosimetry of 111InCHX-DTPA-scFv78-Fc Targeting Endosialin/Tumor Endothelial Marker 1 (TEM1)
Cicone, Francesco, Denoël, Thibaut, Gnesin, Silvano, Riggi, Nicolo, Irving, Melita, Jakka, Gopinadh, Schaefer, Niklaus, Viertl, David, Coukos, George, Prior, John O
Published in Molecular imaging and biology (01.08.2020)
Published in Molecular imaging and biology (01.08.2020)
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Journal Article
Infantile onset myofibrillar myopathy due to recessive CRYAB mutations
Forrest, Katharine M.L, Al-Sarraj, Safa, Sewry, Caroline, Buk, Stefan, Tan, S. Veronica, Pitt, Matthew, Durward, Andrew, McDougall, Marilyn, Irving, Melita, Hanna, Michael G, Matthews, Emma, Sarkozy, Anna, Hudson, Judith, Barresi, Rita, Bushby, Kate, Jungbluth, Heinz, Wraige, Elizabeth
Published in Neuromuscular disorders : NMD (01.01.2011)
Published in Neuromuscular disorders : NMD (01.01.2011)
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Journal Article
Vosoritide treatment accelerates bone growth in children with achondroplasia
Savarirayan, Ravi, Irving, Melita, Hoover-Fong, Julie, Bacino, Carlos A, Ozono, Keiichi, Mohnike, Klaus, Cormier-Daire, Valerie, Leiva-Gea, Antonio, Alanay, Yasemin, Andrews, Mary, Crews, Chandler, Klafehn, Cristina, Jayaram, Kala, Jeha, George S, Fisheleva, Elena, Huntsman-Labed, Alice, Day, Jonathan
Published in Future Rare Diseases (30.09.2021)
Published in Future Rare Diseases (30.09.2021)
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Journal Article
Clinical utility gene card for: 3M syndrome
Holder-Espinasse, Muriel, Irving, Melita, Cormier-Daire, Valérie
Published in European journal of human genetics : EJHG (01.09.2011)
Published in European journal of human genetics : EJHG (01.09.2011)
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Journal Article
Collagen VI glycine mutations: Perturbed assembly and a spectrum of clinical severity
Pace, Rishika A., Peat, Rachel A., Baker, Naomi L., Zamurs, Laura, Mörgelin, Matthias, Irving, Melita, Adams, Naomi E., Bateman, John F., Mowat, David, Smith, Nicholas J. C., Lamont, Phillipa J., Moore, Steven A., Mathews, Katherine D., North, Kathryn N., Lamandé, Shireen R.
Published in Annals of neurology (01.09.2008)
Published in Annals of neurology (01.09.2008)
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Journal Article
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
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
Published in Journal of the Endocrine Society (03.05.2021)
Published in Journal of the Endocrine Society (03.05.2021)
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Journal Article
Possible association between complex congenital heart defects and 11p15 hypomethylation in three patients with severe Silver–Russell syndrome
Ghanim, Mustafa, Rossignol, Sylvie, Delobel, Bruno, Irving, Melita, Miller, Owen, Devisme, Louise, Plennevaux, Jean‐Louis, Lucidarme‐Rossi, Sophie, Manouvrier, Sylvie, Salah, Azzi, Chivu, Olimpia, Netchine, Irène, Vincent‐Delorme, Catherine
Published in American journal of medical genetics. Part A (01.03.2013)
Published in American journal of medical genetics. Part A (01.03.2013)
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