BioMarin、軟骨無形成症に関する疾患啓発ウェブサイト(日本語版)を検討/ BioMarin plans achondroplasia awareness website (Japanese version)

BioMarin、軟骨無形成症に関する疾患啓発ウェブサイト(日本語版)を検討/ BioMarin plans achondroplasia awareness website (Japanese version)

BioMarinは、軟骨無形成症の患者およびご家族、介護者への情報発信を行うべく、米国本社が中心となり疾患啓発サイト(を作成しました。これまでも同社はモルキオ症候群やPKU(フェニルケトン尿症)といった啓発サイトを作成しており、これらには疾患に関わる基本情報からpatient storyに至るまで広範な情報をとりまとめているものです。そして今般の軟骨無形成症のサイト作成に至りました。

一方、疾患啓発サイトの言語が英語のみであることから、日本人がアクセスし難いコンテンツとなっています。こうした言語の問題に加え、医療などを巡る社会制度面や患者個々人の生活面において欧米と大きく異なる日本独自のニーズがあることが想定されます。それらを明確にすべく、上記の英語のウェブサイトをベースに、日本向けの啓発サイトをアジアパシフィックのリージョナルオフィス(香港)の企画に基づき、このほど、BioMarin Japanより調査の依頼がまいりました。















BioMarin has created a disease awareness site ( centered on the US headquarters to disseminate information to patients with achondroplasia, their families, and caregivers. So far, the company has created some websites such as related Morquio Syndrome and PKU, which collect a wide range of information from basic information on diseases to patient stories. And they disclosed the website of achondroplasia at this time.

On the other hand, since the language of the disease awareness site is only written in English, it is difficult for Japanese to access. In addition to the language problem, it seems that there might be unique needs in Japan that are significantly different from those in Europe and the US in terms of social systems related to medical care and the lives of individual patients. In order to clarify them, BioMarin Pharmaceutical Japan has recently requested our collaborations to make it for Japan based on the above English website, initiated its Asia Pacific’s Regional Office (Hong Kong).

[Planning concept]

-Survey method

・ What are the needs of Japanese patients and their families based on qualitative surveys by interviews and quantitative surveys by Google Form? Is there a route to get information about? Clarify what kind of information is missing, and so on.


For families with patients aged 12-18, such as patient association representatives, families with patients aged 0-4, families with patients aged 5-11, etc.

-Disclosure content

・ Although age-specific complications, adaptation measures in daily life, general clinical trial processes, etc. are targeted for creation, basic information on vosoritide and information disclosure up to efficacy/effect are not assumed at the time.

-Publication time

Scheduled to be released next year or later in order to analyze the survey results within the year

[Quantitative survey]

-Response period

Until Wednesday, November 25, 2020

-Time required

Around 10-15 minutes

We have already completed the cooperation of multiple qualitative surveys via GTA, and we would be grateful if you living in Japan could cooperate with us in the quantitative survey within the response period.



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