The World FSHD Alliance, a network of two dozen national patient groups and advocacy organizations for facioscapulohumeral muscular dystrophy (FSHD),

held its fifth annual leadership summit on June 17-18, 2023, in Milan, Italy. Forty in-person and nine virtual delegates from 19 organizations attended the meeting. They share a mission to support patients and speed up the development of treatments for FSHD by:

  • educating, supporting, and activating people with FSHD;
  • finding and engaging clinicians who are interested in providing the best care for patients;
  • working with national registries and supporting research;
  • advocating for patients with their governments, healthcare agencies, insurance, and policymakers.

As drug development has advanced rapidly in the past several years, Alliance members also recognized they need a global strategy of coordination and collaboration to ensure that there are enough “trial-ready” patients and clinical trial sites. Some members are actively working to develop trial infrastructure, genetic testing, and patient registries in countries such as Japan, Brazil, and Israel, that might not otherwise be on the radar for clinical trial sponsors. With the prospect of new treatments coming on the market in the US and EU over the next few years, the Alliance is also discussing country-by country strategies to advocate for patients everywhere to have access to treatments. In response to these emerging challenges, several World FSHD Alliance members proposed a new global initiative called Project Mercury to accelerate trial readiness and ensure widespread access to treatments. This was, appropriately, a focus of this year’s Alliance leadership summit. “In a rare disease like FSHD, patients, expertise, and resources are scarce and precious,” said Mark Stone, CEO of the FSHD Society, who serves as chair of the Project Mercury Global Task Force. “To face the urgent challenges in clinical trials and treatment access, we must bring all stakeholders together in an effective, collaborative, and transparent network. Solutions to the challenges we face require global collaboration, led by the patient advocacy leaders of the World FSHD Alliance.”

For more information about the World FSHD Alliance, visit fshdsociety.org/connect/worldfshdalliance/.

Traduzione Micaela Doni, Gruppo FHSD UILDM.

 

Courtesy of FSHD Society

To stay up to date on FSHD Society activities, you can fill out this form and join the community!

The form is also accessible from the QR Code below.