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…

Vosoritide▼ in children <2 years of age: Real world experience and practical tips

For those of you who were unable to join us for this engaging and informative case-based webinar, the recording is now available on-demand! This webinar aims to provide you with insights from leading clinicians on the practicalities of vosoritide in children <2 years of age, including multidisciplinary team management, neurological considerations within the first year of life, a real-world case study and practical support for parents and caregivers.