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

    Vosoritide treatment accelerates bone growth in children with achondroplasia

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    2021

    Published in: Future Rare Diseases ; 1(2021), 3, Artikel-ID FRD14

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

    Pharmacokinetics and exposure-response of vosoritide in children with achondroplasia

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    2022

    Published in: Clinical pharmacokinetics ; 61(2022), 2, Seite 263-280

  3. Savarirayan, Ravi [Author]; Tofts, Louise [Author]; Irving, Melita [Author]; Wilcox, William [Author]; Bacino, Carlos A. [Author]; Hoover-Fong, Julie [Author]; Font, Rosendo Ullot [Author]; Harmatz, Paul [Author]; Rutsch, Frank [Author]; Bober, Michael B. [Author]; Polgreen, Lynda E. [Author]; Ginebreda, Ignacio [Author]; Mohnike, Klaus [Author]; Charrow, Joel [Author]; Hoernschemeyer, Daniel [Author]; Ozono, Keiichi [Author]; Alanay, Yasemin [Author]; Arundel, Paul [Author]; Kagami, Shoji [Author]; Yasui, Natsuo [Author]; White, Klane K. [Author]; Saal, Howard M. [Author]; Leiva-Gea, Antonio [Author]; Luna-González, Felipe [Author]; [...]

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

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    2021

    Published in: Genetics in medicine ; 23(2021), 12, Seite 2443-2447

  4. Savarirayan, Ravi [Author]; Irving, Melita [Author]; Harmatz, Paul [Author]; Delgado, Borja [Author]; Wilcox, William R. [Author]; Philips, John [Author]; Owen, Natalie [Author]; Bacino, Carlos A. [Author]; Tofts, Louise [Author]; Charrow, Joel [Author]; Polgreen, Lynda E. [Author]; Hoover-Fong, Julie [Author]; Arundel, Paul [Author]; Ginebreda, Ignacio [Author]; Saal, Howard M. [Author]; Basel, Donald [Author]; Font, Rosendo Ullot [Author]; Ozono, Keiichi [Author]; Bober, Michael B. [Author]; Cormier-Daire, Valérie [Author]; Sang, Kim-Hanh Le Quan [Author]; Baujat, Genevieve [Author]; Alanay, Yasemin [Author]; Rutsch, Frank [Author]; [...]

    Growth parameters in children with achondroplasia : a 7-year, prospective, multinational, observational study

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    2022

    Published in: Genetics in medicine ; 24(2022), 12, Seite 2444-2452

  7. Irving, Melita; Savarirayan, Ravi; Arundel, Paul; Polgreen, Lynda; Mohnike, Klaus; Ozono, Keiichi; Saunders, Michael; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan

    eP266: Associations between height and Health-Related Quality of Life (HRQoL) and functional independence in children with achondroplasia

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    Elsevier BV, 2022

    Published in: Genetics in Medicine

  8. 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

    P194: Persistence of growth promoting effects in children with achondroplasia over seven years: Update from phase II extension study with vosoritide*

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    2023

    Published in: Genetics in Medicine Open

  9. Stone, Richard M; Fischer, Thomas; Paquette, Ronald; Schiller, Gary; Schiffer, Charles A.; Ehninger, Gerhard; Cortes, Jorge; Kantarjian, Hagop M.; DeAngelo, Daniel J.; Huntsman-Labed, Alice; Dutreix, Catherine; Rai, Sumita; Giles, Frank

    A Phase 1b Study of Midostaurin (PKC412) in Combination with Daunorubicin and Cytarabine Induction and High-Dose Cytarabine Consolidation in Patients Under Age 61 with Newly Diagnosed De Novo Acute Myeloid Leukemia: Overall Survival of Patients Whose Blasts Have FLT3 Mutations Is Similar to Those with Wild-Type FLT3

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    American Society of Hematology, 2009

    Published in: Blood

  10. 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

    O22: A randomized controlled trial of vosoritide in infants and toddlers with achondroplasia*

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    2023

    Published in: Genetics in Medicine Open

  11. 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

    LBMON196 A Randomized Controlled Trial Of Vosoritide In Infants And Toddlers With Achondroplasia

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    The Endocrine Society, 2022

    Published in: Journal of the Endocrine Society

  12. 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

    Pharmacokinetics and Exposure–Response of Vosoritide in Children with Achondroplasia

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    Springer Science and Business Media LLC, 2022

    Published in: Clinical Pharmacokinetics

  13. Savarirayan, Ravi; Irving, Melita; Bacino, Carlos A.; Bostwick, Bret; Charrow, Joel; Cormier-Daire, Valerie; Le Quan Sang, Kim-Hanh; Dickson, Patricia; Harmatz, Paul; Phillips, John; Owen, Natalie; Cherukuri, Anu; Jayaram, Kala; Jeha, George S.; Larimore, Kevin; Chan, Ming-Liang; Huntsman Labed, Alice; Day, Jonathan; Hoover-Fong, Julie

    C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia

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    Massachusetts Medical Society, 2019

    Published in: New England Journal of Medicine

  14. Gotlib, Jason; Kluin-Nelemans, Hanneke C.; George, Tracy I.; Akin, Cem; Sotlar, Karl; Hermine, Olivier; Awan, Farrukh T.; Hexner, Elizabeth; Mauro, Michael J.; Sternberg, David W.; Villeneuve, Matthieu; Huntsman Labed, Alice; Stanek, Eric J.; Hartmann, Karin; Horny, Hans-Peter; Valent, Peter; Reiter, Andreas

    Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis

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    Massachusetts Medical Society, 2016

    Published in: New England Journal of Medicine

  15. Fischer, Thomas; Stone, Richard M.; DeAngelo, Daniel J.; Galinsky, Ilene; Estey, Elihu; Lanza, Carlo; Fox, Edward; Ehninger, Gerhard; Feldman, Eric J.; Schiller, Gary J.; Klimek, Virginia M.; Nimer, Stephen D.; Gilliland, D. Gary; Dutreix, Catherine; Huntsman-Labed, Alice; Virkus, Jodi; Giles, Francis J.

    Phase IIB Trial of Oral Midostaurin (PKC412), the FMS-Like Tyrosine Kinase 3 Receptor (FLT3) and Multi-Targeted Kinase Inhibitor, in Patients With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome With Either Wild-Type or Mutated FLT3

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    American Society of Clinical Oncology (ASCO), 2010

    Published in: Journal of Clinical Oncology

  16. 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; [...]

    P141: Persistent growth-promoting effects of vosoritide in children with achondroplasia is accompanied by improvement in physical aspects of quality of life*

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    2024

    Published in: Genetics in Medicine Open

  17. 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; [...]

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

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    Elsevier BV, 2021

    Published in: Genetics in Medicine

  18. 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; [...]

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

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    The Endocrine Society, 2020

    Published in: Journal of the Endocrine Society

  19. Savarirayan, Ravi; Irving, Melita; Harmatz, Paul; Delgado, Borja; Wilcox, William R.; Philips, John; Owen, Natalie; Bacino, Carlos A.; Tofts, Louise; Charrow, Joel; Polgreen, Lynda E.; Hoover-Fong, Julie; Arundel, Paul; Ginebreda, Ignacio; Saal, Howard M.; Basel, Donald; Font, Rosendo Ullot; Ozono, Keiichi; Bober, Michael B.; Cormier-Daire, Valerie; Le Quan Sang, Kim-Hanh; Baujat, Genevieve; Alanay, Yasemin; Rutsch, Frank; [...]

    Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study

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    Elsevier BV, 2022

    Published in: Genetics in Medicine