History of FSHD Japan

The Establishment of FSHD Japan

 

Prior to the establishment of FSHD Japan in 2015, the environment surrounding FSHD was as follows.

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First, the Japan Muscular Dystrophy Association managed its members on a regional basis (by prefecture), and there was no membership list categorized by disease type. This was likely because the Japan Muscular Dystrophy Association had a strong character as a regional mutual aid society, with each regional branch operating independently, and there was little perceived need for FSHD patients across the country to communicate with one another.

 

In this context, advances in basic research during the 2010s clarified the complex genetic causes of FSHD and the importance of the DUX4 gene. In other words, the protein targets for FSHD treatments became clear. Naturally, this heightened expectations among FSHD patients for the development of treatments targeting DUX4.

 

However, at the time, there was no patient registration system for FSHD, and there was no way to recruit patients nationwide for clinical trials.It was a natural step to establish a patient registry system for FSHD in advance, in preparation for the possibility of clinical trials for treatment drugs.

 

Furthermore, with the development of the internet, it became easier to access advanced initiatives in Europe and the United States. Japanese FSHD patients first learned about these advanced initiatives, specifically that patient groups specializing in FSHD were taking the lead in treatment drug development by involving medical and pharmaceutical professionals.They realized that simply waiting for doctors and pharmaceutical companies to develop a treatment was not enough; patients must take the initiative to involve medical and pharmaceutical professionals in the development of a treatment in order to obtain it.

 

In light of this situation, there was a growing momentum to establish an FSHD-specific patient association in Japan, and in 2015, the FSHD Japan Subcommittee was established within the Japan Muscular Dystrophy Association.

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Purpose of FSHD Japan's establishment (at the time of establishment)

 

The following were the objectives that FSHD Japan set forth at the time of its establishment in 2015.

Establishment of a patient registry

Development of an information provision system

• Provision of information for early diagnosis (there are no clinical centers for FSHD)

• Provision of information to FSHD patients

Expanding the FSHD community

• Becoming a specialized patient organization for FSHD that can collaborate with overseas FSHD communities

• Engaging with basic/clinical researchers and promoting research

• Building a network to secure support from many people

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Since its establishment in 2015 as the “FSHD Subcommittee” within the Japanese Muscular Dystrophy Association, FSHD Japan has steadily achieved these goals and continues to move forward toward higher objectives.