We are excited to announce that FSHD Finland has joined FSHD Europe. Lauri Pirinen and Lora Chun are the patient representatives of FSHD Finland. They did an incredible job in starting their own organization in a very short time. We look forward to a successful collaboration.
Curious about their inspiring story? In a case study, they share how FSHD Finland was established, and it provides valuable insights to encourage other countries to start their own patient organization.
FSHD Europe participated in the 279th ENMC workshop “Classification, clinical care, outcome measures and biomarkers in childhood-onset FSHD: towards standardising clinical care and ensuring clinical trial readiness”. Twenty-seven participants, including clinicians, research experts, patient advocates, and industry representatives, from 14 different countries engaged in this workshop.
FSHD Europe would like to thank the organizers, Dr C. Erasmus (The Netherlands), Prof. K. Mathews (USA), K. de Valle (Australia) and Prof. T. Willis (UK), for the opportunity to present on the FSHD European Trial Network & Market Access. We had very interesting and fruitful discussions on the spectrum of FSHD, the challenges of trials in children, and the most appropriate clinical outcome measures. Gaps of knowledge were defined, and task groups were formed to work on this. Hence, we will continue working together towards clinical trial readiness for children with FSHD.
For more information, please read the lay report which is published on the ENMC website (click here).
Lay reports – ENMC
Save the date: FSHD Connect Europe patient meeting 2025
FSHD Europe will organize, in collaboration with the FSHD Society, the first FSHD Connect Europe meeting on June 13th – June 15th, 2025, in Amsterdam, The Netherlands.
NEW date: 32nd FSHD International Research Congress, June 12th – 13th, 2025, Amsterdam, The Netherlands
FSHD Society’s annual FSHD International Research Congress (IRC) is the premier global conference exclusively focused on facioscapulohumeral muscular dystrophy (FSHD) research.
FSHD Europe came together this weekend in France for our General
Assembly Meeting. First, we had a wonderful tour at the laboratory of hashtag#Généthon / hashtag#AFMThéléthon in Evry and learned about gene therapy
development for rare diseases. During the following days, patient representatives of our 13 member organisations came together. We had a very productive and enjoyable meeting where we recognized how far we have come, identified challenges and opportunities, set our strategy, and defined activities to realise our goals. Thank you to all participants for this great weekend! FSHD Europe is the united voice of people with FSHD.
FSHD Europe congratulate Nicol Voermans with the appointment as Professor of Muscular Diseases at Radboud University, Nijmegen, the Netherlands. On Thursday September 12th, Nicol delivered her inspirational inaugural speech entitled: ‘Between Hype and Hope’.
One of the next steps on our journey is to engage with regulators to understand the process of approving drugs to be available to patients across Europe. Therefore, FSHD Europe organized a webinar with EMA (European Medicine Agency) to learn more about the role of patients in their approval process. It was an informative webinar, and we would like to thank the speaker, Maria Mavris (Patient Liaison, EMA) and all the patient representatives attending.
Recently two national patient organizations, FSHD UK and MD Slovenia joined FSHD Europe as new member organizations. FSHD Europe is very pleased having now 12 members from 10 different countries (Sweden, France, UK, Germany, Netherlands, Italy, Spain, Denmark, Slovenia, and Belgium). The aim is to include more European countries. If you know national patient organizations or patient groups involved in FSHD, not yet connected to FSHD Europe. Please refer them to Ria de Haas (ria@fshd-europe.info).
On September 12th, 2024, Fulcrum announced that its Phase 3 REACH trial evaluating losmapimod in patients with FSHD, did not achieve its primary endpoint of change from baseline in RSA (measure of reachable workspace) with losmapimod compared to placebo. In addition, secondary endpoints did not achieve nominal statistical significance. The company decided to suspend its development of Losmapimod.
The company Avidity shared a press release to announce initial AOC 1020 data from the Phase 1/2 FORTITUDE™ trial in adults living with FSHD.
The first World FSHD Day was celebrated on June 20, 2016. Three years earlier, brothers Marco and Sandro Biviano traveled from their home on Lipari, Italy to Rome to advocate for better access to medical care.
Next year the IRC will take place in Amsterdam, the Netherlands on June 19-20, 2025. Furthermore, FSHD Europe is aiming to organize the first European FSHD Connect Meeting in collaboration with the FSHD Society on June 21-22, 2025, in Amsterdam.