{"id":3539,"date":"2024-02-04T17:13:24","date_gmt":"2024-02-04T17:13:24","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-gb\/achondroplasia\/?page_id=3539"},"modified":"2025-09-08T14:46:54","modified_gmt":"2025-09-08T14:46:54","slug":"achondroplasia-expert-home","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-gb\/achondroplasia\/expert\/","title":{"rendered":"Achondroplasia Expert Home"},"content":{"rendered":"
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Welcome to Achondroplasia Expert<\/h2>\n

We provide free educational resources for healthcare professionals practicing across Europe, Canada, Australia and New Zealand to facilitate improvement in clinical outcomes and quality of life for children and adults with achondroplasia.<\/p>\n

This website may contain promotional material on BioMarin products, which is displayed based on the prescribing information approved by the European Medicines Agency \u2013 EMA.<\/p>\n <\/div>\n \n

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Please be aware that marketing authorization for BioMarin products may vary from country to country, and where products are registered, they might have different labels as approved by the local regulatory agencies. Please consult with BioMarin Medical Information<\/a> for more information.<\/p>\n

Get access to the latest developments in achondroplasia, and keep yourself up to date with publication summaries, congress reviews, podcasts, and other educational videos. Register today and join a network of specialists dedicated to the management of the disease.<\/p>\n

Register or log in now<\/a><\/p>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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The Vosoritide\u25bc<\/span>: A year of data webinar is now available to view on demand! This virtual event shares a review of the key vosoritide data presented in 2024, alongside a look ahead at what to expect in 2025. The webinar concluded with a discussion of what these updates mean for clinical practice.<\/span><\/p>\n

Watch now<\/a><\/p>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Latest updates\n<\/h2>\n <\/div>\n\t\t\t\t\t\t \n \n \n \n \n
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Achondroplasia.expert literature review: January\u2013March publications 2025<\/span>\n<\/h2>\n

Six papers were highlighted in our review of the literature from the first quarter of 2025. Barreda-Bonis et al. <\/em>presented a protocol developed via expert consensus for the administration of vosoritide\u25bc<\/span> in Spain. A manuscript by Cormier-Daire et al.<\/em> shared data from a vosoritide real-world early access program in France which indicated a positive benefit\u2013risk profile consistent with clinical trial data. Hoover-Fong et al. <\/em>presented a guide for HCPs conducting anthropometric measurements of people with disproportionate short stature. Full deck summaries are also available for these three publications.<\/p>\n

Merchant, Hoover-Fong, and Carroll presented 4 scenarios illustrating age-specific ACH-related issues that an endocrinologist may face in a clinical setting when considering treatment with vosoritide. Reincke et al<\/em>. shared the real-world data on growth in children with ACH treated with vosoritide over a 12-month period. Finally, Vallin et al.<\/em> carried out a study to describe foetal imaging and the ante- and postnatal management of pregnancies diagnosed with foetal ACH.<\/p>\n <\/div>\n\n

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International consensus guidelines on vosoritide\u25bc\u00a0in with achondroplasia\n<\/h2>\n

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.<\/p>\n

Access via PubMed<\/a><\/p>\n <\/div>\n\n

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Vosoritide\u25bc\u00a0and health-related quality of life in children with achondroplasia\n<\/h2>\n

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 \u2265 1 SD increase in height Z-score\u2026<\/p>\n <\/div>\n\n

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Publications<\/h4>\n

Free slide summaries for a selection of papers <\/strong>
\nThe Editorial Committee regularly select the most relevant and up-to-date publications for review, with slide summaries of the papers available to download.<\/p>\n \n <\/div>\n

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View all Publications <\/a><\/p>\n <\/div>\n<\/div>\n\n

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Educational Content<\/h4>\n

Free interactive videos, tutorials and assessments <\/strong>
\nA range of distance learning programmes to meet the educational need of healthcare professionals in achondroplasia. Key topics of interest have been identified through consultation and guidance from our Educational Committee.<\/p>\n \n <\/div>\n

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View all Educational Content<\/a><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Podcasts<\/h3>\n

Freely accessible podcasts<\/strong>
\nWorld leading experts discussing a wide variety of topics in achondroplasia. <\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

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Listen to our podcasts <\/a><\/p>\n <\/div>\n\t\t\t\t\t <\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Useful Links\n<\/h2>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t
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Associations<\/h3>\n
    \n
  1. ENDO<\/a><\/li>\n
  2. EPOS<\/a><\/li>\n
  3. ACMG<\/a><\/li>\n
  4. ESPE<\/a><\/li>\n
  5. PES<\/a><\/li>\n
  6. JSPE<\/a><\/li>\n
  7. ISDS<\/a><\/li>\n<\/ol>\n <\/div>\n<\/div>\n\n
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    Upcoming Events<\/h3>\n
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    1. ACMG 2025<\/a><\/li>\n
    2. ESPE and ESE 2025<\/a><\/li>\n
    3. JSPE 2025<\/a><\/li>\n<\/ol>\n <\/div>\n<\/div>\n\n
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      BioMarin<\/h3>\n
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      1. hcp.achondroplasia.com<\/a><\/li>\n
      2. Achondroplasia.com<\/a><\/li>\n
      3. BioMarin.com<\/a><\/li>\n<\/ol>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":4,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-3539","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nAchondroplasia Expert Home - BioMarin HCP<\/title>\n<meta name=\"description\" content=\"Discover expert insights and resources on achondroplasia at BioMarin. 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