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Topics

Learn about achondroplasia topics

Real World Evidence

Publication

Topics

Real World Evidence

Changes in the alignment of the spine and lower limb in children with achondroplasia treated with Vosoritide: A single-center, 1-year follow-up prospective study

Real-world safety and effectiveness of Vosoritide in children with achondroplasia: French early access program

Real-world safety and effectiveness of Vosoritide in achondroplasia: Results from a single center in Portugal

Real-world outcome of Vosoritide treatment in children with achondroplasia: A 12-month retrospective observational study

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

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

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

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

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

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

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

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

Impact of Disease

Anthropometric measurements guide in disproportionate short stature

Achondroplasia and Hypochondroplasia in France: a nationwide epidemiological analysis

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

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

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)

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

Health Supervision for People With Achondroplasia Summary Table

Examining the disability paradox and health state utility valuation in achondroplasia

Achondroplasia Growth Curves

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

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

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

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

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

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

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

Potential Achondroplasia Complications by Age Group

Mechanism of Action

Achondroplasia Mechanism of Action

Safety and Efficacy Profile

Changes in the alignment of the spine and lower limb in children with achondroplasia treated with Vosoritide: A single-center, 1-year follow-up prospective study

Real-world safety and effectiveness of Vosoritide in children with achondroplasia: French early access program

Real-world safety and effectiveness of Vosoritide in achondroplasia: Results from a single center in Portugal

Real-world outcome of Vosoritide treatment in children with achondroplasia: A 12-month retrospective observational study

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

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

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

Vosoritide improves tibial bowing in infants and toddlers with achondroplasia

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

Vosoritide improves tibial bowing in infants and toddlers with achondroplasia

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

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

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

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

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

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

Does Vosoritide Treatment Affect Bone Strength in Children with Achondroplasia?

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

Vosoritide for Children with Achondroplasia: Growth Velocity and Pubertal Milestones

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

Vosoritide Treatment Accelerates Bone Growth in Children with Achondroplasia

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