International consensus guidelines on vosoritide▼ in with achondroplasia

This study was carried out to provide practical guidance for professionals and health services worldwide on vosoritide use in the treatment of achondroplasia. A total of 16 leading experts and patient advocates representing nine countries convened to form a guideline development group. This group developed statements to provide recommendations across the treatment pathway, from treatment initiation, ongoing monitoring and evaluation, to stopping vosoritide treatment and the continued follow up after cessation. These guidelines recommend a minimum set of requirements and a practical framework for professionals and health services worldwide.

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Vosoritide▼ and health-related quality of life in children with achondroplasia

Savarirayan et al. assessed the impact of vosoritide on growth-associated health-related quality of life (HRQoL) in children with achondroplasia. Data were gathered using the Quality of Life in Short Stature Youth (QoLISSY) questionnaire. Vosoritide improved self-reported and caregiver-reported QoLISSY physical and social scores, with the greatest increases being seen in participants with a ≥ 1 SD increase in height Z-score…

Growth-promoting effects of vosoritide▼ in children with achondroplasia

Savarirayan et al. reported on data from the 111-302 open label extension study, with the aim of exploring the long-term safety, tolerability, and efficacy of vosoritide treatment in children with achondroplasia. Vosoritide treatment was found to be well tolerated and resulted in sustained growth promoting effects in children with achondroplasia who were treated with daily subcutaneous injections of vosoritide for up to 6 years.

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