News

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).

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 20th – June 22nd, 2025, in Amsterdam, The Netherlands.

Save the date: 32nd FSHD International Research Congress, June 19th – 20th, 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.

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.

Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules, today announced that it has entered into a collaboration and license agreement with Sanofi (Nasdaq: SNY) for the development and commercialization of losmapimod, an oral small molecule being investigated for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

In 2012, a standard procedure was established for the genetic diagnosis of facioscapulohumeral muscular dystrophy (FSHD). Due to the increasing complexity of FSHD genetics, the rise in genetic testing centers, and the start of clinical trials for FSHD, an update on the genetic features of FSHD and international testing guidelines is needed.