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• Achondroplasia
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2025

Clinical Expert Opinion on the Impact of Vosoritide on Health and Function in Children With Achondroplasia

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• Congresses
• Achondroplasia
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2025

Socio-Economic Burden Among Children and Adults With Achondroplasia in Europe and Latin America

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• Congresses
• Achondroplasia
• Hypochondroplasia
• ESPE-OSCAR Science Symposium
• 2025
• Impact of Disease

Achondroplasia and Hypochondroplasia in France: a nationwide epidemiological analysis

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• Congresses
• Noonan syndrome
• SHOX deficiency
• Turner syndrome
• American Society for Bone & Mineral Research (ASBMR)
• 2025

Criteria for defining inadequate response to growth hormone in Noonan syndrome, Turner syndrome, and SHOX deficiency

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• Congresses
• Achondroplasia
• American Society for Bone & Mineral Research (ASBMR)
• 2025
• Safety and Efficacy Profile

Effect of vosoritide on spine morphology in young children with achondroplasia: 1-year results from a double-blind, randomized phase 2 study

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• Congresses
• Achondroplasia
• American Society for Bone & Mineral Research (ASBMR)
• 2025
• Safety and Efficacy Profile

Continued growth after puberty in participants with achondroplasia treated with vosoritide in a phase 3 long-term extension trial

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• Congresses
• Achondroplasia
• American Society for Bone & Mineral Research (ASBMR)
• 2025
• Safety and Efficacy Profile

Effect of long-term vosoritide treatment in pediatric participants with achondroplasia on bone mineral density and bone content: results from quantitative computed tomography analyses

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• Congresses
• Achondroplasia
• Human Genetics Society of Australasia (HGSA)
• 2025
• Safety and Efficacy Profile

Vosoritide improves tibial bowing in infants and toddlers with achondroplasia

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• Congresses
• Achondroplasia
• Japan Endocrine Society (JES)
• 2025
• Real World Evidence
• Safety and Efficacy Profile

Real-world safety of vosoritide in children with achondroplasia in Japan

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2025
• Safety and Efficacy Profile

Vosoritide improves tibial bowing in infants and toddlers with achondroplasia

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• Congresses
• Turner syndrome
• Pediatric Endocrine Society (PES)
• 2025

Preliminary Assessment of Vosoritide for Short Stature in Turner Syndrome: 6-month data from a Clinical Trial

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Presentation

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• European Society of Endocrinology (ESE)
• 2025
• Safety and Efficacy Profile

Long-term height gain and maintenance of treatment effect in children with achondroplasia receiving vosoritide

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• Idiopathic Short Stature (ISS)
• European Society for Paediatric Endocrinology (ESPE)
• European Society of Endocrinology (ESE)
• 2025

Design of a phase 2, randomized, controlled, multicentre study of vosoritide treatment in children with idiopathic short stature

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• Congresses
• Noonan syndrome
• SHOX deficiency
• Turner syndrome
• European Society for Paediatric Endocrinology (ESPE)
• European Society of Endocrinology (ESE)
• 2025

Design of a randomized, multicentre, phase 2 study of vosoritide in children with Turner syndrome, Noonan syndrome, or short stature homeobox-containing gene (SHOX) deficiency

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• European Society of Endocrinology (ESE)
• 2025

The Achondroplasia Roadmap: Connecting With the Community

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2025

Interdisciplinary Variant Re-Classification: FGFR3 as an example of genotypic investigation in suspected skeletal dysplasia population

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• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2025
• Impact of Disease

How helpful are sleep studies in determining surgical need in infants with achondroplasia?

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2025

Beyond Boundaries: The Continuous Spectrum in FGFR3-Related Conditions

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2025
• Real World Evidence
• Safety and Efficacy Profile

Safety profile, effectiveness, and adherence of vosoritide in young children with achondroplasia in Japan

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Presentation

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2024
• Real World Evidence
• Safety and Efficacy Profile

Expansion of the CrescNet Registry Achondroplasia Module: Real-World Demographic Data and Outcomes after up to 2 Years of Vosoritide Treatment

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2024

Comparison of the diagnostic yield of whole exome sequencing (WES) and targeted panel sequencing for children with idiopathic short stature (ISS)

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2024
• Real World Evidence
• Safety and Efficacy Profile

Real-world effectiveness of vosoritide in children with achondroplasia: Results from 18 months follow-up in France

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Poster

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• Congresses
• Achondroplasia
• The Professional Society for Health Economics & Outcomes Research Europe (ISPOR-EU)
• 2024
• Impact of Disease

Measuring the caregiver burden in children, adolescents and young adults with Achondroplasia: Results from a prospective, cross-sectional, multi-country study

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2024
• Impact of Disease

Patient-Centred Data Collection Provides Comprehensive Insights into Healthcare Resource Use in Achondroplasia: Data From the Pilot Phase of the VIrtual STudy in Achondroplasia (VISTA)

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Presentation

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• Congresses
• Achondroplasia
• Japanese Society for Pediatric Endocrinology (JSPE)
• 2024
• Safety and Efficacy Profile

Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of their of Quality of Life

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Presentation

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• Congresses
• Achondroplasia
• Japanese Society for Pediatric Endocrinology (JSPE)
• 2024
• Safety and Efficacy Profile

Persistence of Growth Promoting Effects in Children with Achondroplasia Up to 7 Years: Update from a Phase 2 Extension Study with Vosoritide

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Poster

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 2024

Design and objectives of the Acorn study: a non-interventional study evaluating long-term safety in achondroplasia children treated with vosoritide

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 2024

The Achondroplasia Roadmap

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 2024
• Safety and Efficacy Profile

Does Vosoritide Treatment Affect Bone Strength in Children with Achondroplasia?

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 2024
• Impact of Disease

Caregiver perspectives on vosoritide treatment: meaningful HRQoL improvements in children with achondroplasia

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• Congresses
• Achondroplasia
• Congresso Brasileiro de Genética Médica (CBGM)
• 2024
• Safety and Efficacy Profile

Growth-promoting effects of vosoritide in children with achondroplasia aged ≥ 10 years at treatment initiation: results from a Phase 3 extension study

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Presentation

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• Congresses
• Achondroplasia
• Congresso Brasileiro de Genética Médica (CBGM)
• 2024
• Safety and Efficacy Profile

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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• Congresses
• Achondroplasia
• Congresso Brasileiro de Genética Médica (CBGM)
• 2024
• Safety and Efficacy Profile

Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 4 years: Update from Phase 3 extension study

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Presentation

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• Congresses
• Achondroplasia
• International Conference on Children’s Bone Health (ICCBH)
• 2024
• Safety and Efficacy Profile

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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• Congresses
• Achondroplasia
• International Conference on Children’s Bone Health (ICCBH)
• 2024
• Safety and Efficacy Profile

Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 4 years: Update from Phase 3 extension study

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Poster

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• Congresses
• Achondroplasia
• International Conference on Children’s Bone Health (ICCBH)
• 2024
• Safety and Efficacy Profile

Persistence of growth promoting effects in infants and toddlers with achondroplasia: Results from a phase II extension study with vosoritide

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Poster

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• Congresses
• Achondroplasia
• International Conference on Children’s Bone Health (ICCBH)
• 2024

Design and objectives of the Acorn study: a non-interventional study evaluating long-term safety in achondroplasia children treated with vosoritide

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Poster

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• Congresses
• Achondroplasia
• European Human Genetics Conference (ESHG)
• 2024
• Real World Evidence
• Safety and Efficacy Profile

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

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• Congresses
• Achondroplasia
• The Professional Society for Health Economics & Outcomes Research (ISPOR)
• 2024
• Impact of Disease

Examining the disability paradox and health state utility valuation in achondroplasia

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Presentation

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2024
• Safety and Efficacy Profile

A Prospective Trial of Vosoritide in Selected Genetic Causes of Short Stature

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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2024
• Safety and Efficacy Profile

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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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2024
• Safety and Efficacy Profile

Persistent growth-promoting effects of vosoritide in children with achondroplasia for up to 4 years: update from Phase 3 extension study

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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2024
• Safety and Efficacy Profile

Persistence of growth promoting effects in infants and toddlers with achondroplasia: Results from a phase II extension study with vosoritide

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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2024

Growth-promoting effects of vosoritide in children with achondroplasia aged ≥10 years at treatment initiation: results from a phase 3 extension study

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Poster

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• Congresses
• Achondroplasia
• International Pediatric Sleep Association (IPSA)
• 2024
• Safety and Efficacy Profile

Impact of vosoritide on polysomnography parameters among children aged 3-59 months

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2024
• Safety and Efficacy Profile

Persistence of growth-promoting effects in children with achondroplasia up to 7 years: update from a phase 2 extension study with vosoritide

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2024
• Safety and Efficacy Profile

Persistence of growth promoting effects in infants and toddlers with achondroplasia: Results from a Phase II extension study with vosoritide

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2024
• Safety and Efficacy Profile

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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Presentation

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2023

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

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2023
• Real World Evidence
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Real World Evidence

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Impact of Disease

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Impact of Disease

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Genetic Testing & Diagnosis

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2023
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2022
• Real World Evidence
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 2022
• Safety and Efficacy Profile

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

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Presentation

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• Congresses
• Achondroplasia
• International Skeletal Dysplasia Society (ISDS)
• 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

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Poster

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• Congresses
• Achondroplasia
• Endocrine Society (ENDO)
• 2022
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• Pediatric Endocrine Society (PES)
• 2022
• Safety and Efficacy Profile

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

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Presentation

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2022
• Safety and Efficacy Profile

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2022
• Impact of Disease
• Safety and Efficacy Profile

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2022
• Impact of Disease

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2022
• Impact of Disease

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

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Poster

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• Congresses
• Achondroplasia
• American College of Medical Genetics & Genomics (ACMG)
• 2022
• Genetic Testing & Diagnosis

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

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2021
• Impact of Disease

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

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2021

Vosoritide Clinical Study Data Demonstrates CXM is a Superior Biomarker of Endochondral Bone Growth

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Poster

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• Congresses
• Achondroplasia
• European Society for Paediatric Endocrinology (ESPE)
• 2021
• Impact of Disease

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

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