Leaders from patient advocacy groups from around the world meet to advance clinical trial readiness and access to treatments for facioscapulohumeral muscular dystrophy
RANDOLPH, Mass., June 19, 2023 /PRNewswire-PRWeb/ -- The World FSHD Alliance, a network of some two dozen national patient groups and advocacy organizations for facioscapulohumeral muscular dystrophy (FSHD), convened its fifth annual leadership summit this past weekend in Milan, Italy. The delegates share a mission to support patients and speed up the development of treatments for FSHD, a rare, hereditary muscle-weakening condition that affects an estimated one million men, women, and children worldwide. It is marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability.
There is currently no medication to slow or halt the progression of symptoms, but drug development has advanced rapidly in the past several years. There are now three active, multi-center, multinational clinical trials for FSHD, with growing numbers of therapeutic candidates likely to begin clinical trials over the next 2-3 years.
The Alliance members believe that a global strategy of coordination and cooperation is needed to ensure that there are enough "trial-ready" patients and clinical trial sites. Some members are working to develop trial infrastructure and patient registries in countries that might not otherwise be on the radar for clinical trial sponsors. With the prospect of new treatments coming on the market in the U.S. 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.
This year's leadership summit focused on Project Mercury, a new global initiative proposed by several World FSHD Alliance members and governed by multiple patient advocacy groups. The project is building a network of diverse stakeholders, including industry, foundations, and key opinion leaders to collaborate on accelerating clinical trial readiness, patient recruitment, and advocacy vis a vis healthcare regulators and payors.
"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 Project Mercury's 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."
Founding members of Project Mercury's Global Task Force include: FSHD UK, FSHD Canada Foundation, FSHD Europe, FSHD Global Research Foundation, FSHD Society, FSHD Stichting, Avidity Biosciences, Fulcrum Therapeutics, JZ Zebra Consulting, University of Calgary, EURO-NMD, FSHD European Trial Network, and TREAT-NMD.
About the World FSHD Alliance
Founded in 2019, the World FSHD Alliance is an informal association of patient advocacy groups for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy. Alliance members share resources and strategy to provide effective patient education, support, and advocacy. Because FSHD clinical trials will be international in scope, the Alliance also coordinates messaging and outreach efforts for timely, cost-effective engagement with industry around drug development, clinical trials, regulatory approval, and patients' access to therapies.
Media Contact
June Kinoshita, FSHD Society, 7813016060, [email protected]
SOURCE World FSHD Alliance
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