International FSHD Patient Advocacy Summit

Marseille meeting convenes delegates from 11 nations

BY SHEILA HAWKINS, TRUSTEE, MUSCULAR DYSTROPHY UK

FSH muscular dystrophy affects people of every nation and ethnicity, and the effort to develop treatments will undoubtedly require the participation of patients from multiple countries. With international clinical trials already under way and more on the horizon, it was more than timely this June 18 when patient advocates from 13 organizations and 11 nations gathered in Marseille for the first-ever International FSHD Patient Advocacy Summit to discuss global collaboration and coordination.

The conference was sponsored by the FSHD Society and jointly organized with FSHD Europe. It was attended by 38 delegates from Brazil, China, France, Germany, Israel, Italy, Japan, the Netherlands, Spain, UK and the US. More than half of the delegates were patients and family members, some of whom are also doctors or researchers in the FSHD field, and the others were scientists or representatives of the FSHD Society.

It was important for the attendees to make connections, so we got to talking to one another early in the day.  Hearing about patient groups in the different countries was fascinating. It was sobering to learn that in some countries genetic testing was either unavailable or so expensive that few people could afford it.

We heard about the latest research from Jeffrey Statland, Doris Leung, and Scott Harper, and looked at how we could develop strategies for patient engagement, working with scientists and industry, and fundraising. 

We also discussed the concept of a global “contact registry” or patient database so that people around the world could be educated about FSHD research studies and drug trials and be contacted when a study is enrolling patients in specific geographic regions. A key benefit of such a database is that it enables the advocacy groups to work more effectively with companies to coordinate study recruitment campaigns globally.

One issue that will need to be solved is how such a contact registry would comply with Europe’s General Data Protection Regulation, which governs how individuals consent to provide and share their personal information. Even when this is resolved, some patients may feel they already receive enough information to participate in trials in their own country, and might not see the need to join a global list.

What was most exciting about this event was the opportunity to meet people from all over the world with FSHD, to see that although their symptoms might be similar, the healthcare systems and cultural attitudes toward disability make such a difference as to how they are able to get on with their lives. 

Building an international FSHD patient community will mean understanding and recognizing these differences and developing local approaches to patient engagement.

Note: It has been 30 years since Sheila Hawkins was diagnosed with FSHD. She has found it very helpful to be part of the FSH-MD Support Group in the UK. Hawkins is a trustee of Muscular Dystrophy UK and represents the UK FSH-MD Support Group in FSHD Europe, a collaboration of patient groups from six different countries across Europe. 

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