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Achondroplasia

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Achondroplasia Congress Posters and Presentations

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Presentation

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2023

Persistence of Growth Promoting Effects in Infants and Toddlers with Achondroplasia: Results in Children Aged Over 2 Years Old from a Phase II Extension Study with Vosoritide

Presenting Author: M. Irving

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2023

Objectives and Design of the Acorn Study: A Non-Interventional Study Evaluating Long-term Safety in Achondroplasia Patients Treated with Vosoritide

J. M. Pimenta, S. Cohen, S. Mukherjee, F. Fettes, K. Jayaram, E. Lausch

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2023

Real-world safety and effectiveness of vosoritide: Results from an early access program in France

V. Cormier-Daire, T. Edouard, B. Isidor, S. Cohen, S. Mukherjee, J. Pimenta, L. Lhaneche, M. Rossi, E. Schaefer, E. Goodman, S. Sigaudy, G. Baujat

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Poster

  • Congresses
  • Achondroplasia
  • Pediatric Endocrine Society
  • 2023

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

L. E. Polgreen, J. Hoover-Fong, M. Irving, C. A. Bacino, J. Charrow, V. Cormier-Daire, P. Harmatz, A. Huntsman-Labed, E. Fisheleva, I. Sabir, J. Day, J. Phillips, R. Savarirayan

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Persistence of Growth Promoting Effects in Children with Achondroplasia Over Seven Years: Update from Phase II Extension Study with Vosoritide

J. Hoover-Fong, M. Irving, C. A. Bacino, J. Charrow, V. Cormier-Daire, L. E. Polgreen, P. Harmatz, A. Huntsman-Labed, E. Fisheleva, I. Sabir, J. Day, J. Phillips, R. Savarirayan

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Objectives and design of the Acorn Study: A non-interventional study evaluating long-term safety in achondroplasia patients treated with vosoritide

J. M. Pimenta, S. Cohen, S. Mukherjee, F. Fettes, K. Jayaram, E. Lausch

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice

O. Semler, V. Cormier-Daire, E. Lausch, M. B. Bober, R. Carroll, S. B. Sousa, D. Deyle, M. Faden, G. Hartmann, A. Huser, J. Legare, K. Mohnike, T. Rohrer, F. Rutsch, P. Smith, A. Travessa, A. Verado, K. White, W. Wilcox, J. Hoover-Fong

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Quantification of the surgical burden associated with achondroplasia: a comprehensive review of the literature

M. Stender, J. Pimenta, M. Irving, S. Mukherjee

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Health-related quality of life (HRQoL) in achondroplasia: findings from LISA (Life Impact Study on Achondroplasia), a multinational and observational study in Latin America

J. C. Llerena Jr., V. Fano, P. Rosselli, M. Del Pino, C. Valenzuela, J. Méndez, N. C. Cavalcanti, P. A. Thomazinho, A. Aragão, J. Thomaz, T. Butt, R. Rowell, J. Pimenta, S. Cohen, R. Shediac, T. Magalhães, D. Bertola, C. A. Kim

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Gene panels for skeletal dysplasia and epilepsy: Maximizing clinical utility through careful design, regular review, and clinician-laboratory collaboration

A. A. Tayoun, S. Aradhya, M. Bailey, L. Bastaki, S. Došenović, F. Ezgü, E. Izzo, C. Lampe, J. J. O’Byrne, G. Seratti, C. Snow, C. Staufner, P. Striano

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics and Genomics
  • 2023

Persistent and stable growth promoting effects of vosoritide in children with achondroplasia for up to 3.5 years: results from an ongoing Phase 3 extension study

J. Hoover-Fong, R. Savarirayan, L Tofts, M. Irving, W. R. Wilcox, C. A. Bacino, R. U. Font, P. Harmatz, F. Rutsch, M. B. Bober, L. E. Polgreen, I. Ginebreda, K. Mohnike, J. Charrow, D. Hoernschemeyer, K. Ozono, Y. Alanay, P. Arundel, S. Kagami, N. Yasui, K. White, H. M. Saal, A. Leiva-Gea, F. Luna-González, H. Mochizuki, D. Basel, D. M. Porco, K. Jayaram, E. Fisheleva, S. Lawrinson, J. Day

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2022

Real-world experience with vosoritide for achondroplasia: Interim findings from an early access programme in France

V. Cormier-Daire1, S. Cohen, T. Edouard, B. Isidor, S. Mukherjee, J. Pimenta, M. Rossi, E. Schaefer, S. Sigaudy and G. Baujat

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Poster

  • Congresses
  • Achondroplasia
  • International Skeletal Dysplasia Society
  • 2022

Persistent growth in children with achondroplasia treated with vosoritide for two years: further evidence supporting the first precision therapy for this condition

R. Savarirayan, L. Tofts, M. Irving, W. Wilcox, C. A. Bacino, J. Hoover-Fong, R. Ullot Font, P. Harmatz, F. Rutsch, M. B. Bober, L. E. Polgreen, I. Ginebreda, K. Mohnike, J. Charrow, D. Hoernschemeyer, K. Ozono, Y. Alanay, P. Arundel, S. Kagami, N. Yasui, K. White, H. M. Saal, A. Leiva-Gea, F. Luna-González, H. Mochizuki, D. Basel, D. M. Porco, K. Jayaram, E. Fisheleva, L. Han, J. Day

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Poster

  • Congresses
  • Achondroplasia
  • International Skeletal Dysplasia Society
  • 2022

Health related quality of life (HRQoL) in Achondroplasia: findings from LISA (Life Impact Study on Achondroplasia), a multinational and observational study in Latin America

Dr. J. Llerena Jr.

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Poster

  • Congresses
  • Achondroplasia
  • Endocrine Society
  • 2022

A Randomized Controlled Trial of Vosoritide in Infants and Toddlers with Achondroplasia

R. Savarirayan, W. Wilcox, Paul Harmatz, J. Phillips III, L. Polgreen, L. Tofts, K. Ozono, P. Arundel, M. Irving, C. Bacino, D. Basel, M. Bober, J. Charrow, H. Mochizuki, Y. Kotani, H. Saal, G. Jeha, L. Han, E. Fisheleva, A. Huntsman-Labed, J. Day

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Poster

  • Congresses
  • Achondroplasia
  • Pediatric Endocrine Society
  • 2022

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

L. Polgreen

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Presentation

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics & Genomics

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

Presenting Author: M. Irving

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics & Genomics
  • 2022

Expert opinions regarding impact of achondroplasia on health-related quality of life and long-term effects of vosoritide: a modified delphi study

R. Savarirayan, W. Baratela, T. Butt, V. Cormier-Daire, M. Irving, B. Miller, K. Mohnike, K. Ozono, R. Rosenfeld, A. Selicorni, D. Thompson, K. White, M. Wright, S. Fredwall

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics & Genomics
  • 2022

Higher rates of non-skeletal complications in achondroplasia compared to the general population: a UK matched cohort study using CPRD-GOLD database

M. Irving, J. Pimenta, M. Cheung, L. MazzeoL, S. Landis, S. Mukherjee

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics & Genomics
  • 2022

Associations between height and health-related quality of life (HRQoL) and functional independence in children with achondroplasia

M. Irving, R. Savarirayan, P.Arundel, L. Polgreen, K. Mohnike, K. Ozono, M. Saunders, E. Fisheleva, A. Huntsman-Labed, J. Day

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Poster

  • Congresses
  • Achondroplasia
  • American College of Medical Genetics & Genomics
  • 2022

Clinical utility of a sponsored, no-charge skeletal dysplasia gene panel testing program: Results from >2600 tests

G. MacCarrick, W. Mackenzie, C. Raggio, K. White, D. Krakow, E. Izzo, V. Pansare, A. Hunt, R. Shediac, D. Chu, T. Yar Pang, S. Poll , H. McLaughlin, G. Seratti

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2021

Higher Rates of Non-skeletal Complications in Achondroplasia Compared to the General Population: A UK matched cohort study using the CPRD database

M. Irving, J. Pimenta, M. Cheung, L. Mazzeo, S. Landis, S. Mukherjee

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2021

Vosoritide clinical study data demonstrates CXM is a superior biomarker of endochondral bone growth

K. Larimore, T. Nguyen, Y. Qi, G. Jeha, S. Zoog

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Poster

  • Congresses
  • Achondroplasia
  • European Society for Paediatric Endocrinology
  • 2021

Associations between height and health-related quality of life (HRQoL) and functional independence in children with achondroplasia

M. Irving, R. Savarirayan, P. Arundel, L. Polgreen, K. Mohnike, K. Ozono, M. Saunders, E. Fisheleva, A. Huntsman-Labed, J. Day

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