News

Six months into the PaLaDIn project, FSHD Europe’s contribution to Work Package 9 is progressing well and remains on track. Funded by the Innovative Health Initiative (IHI), PaLaDIn—Patient Lifestyle and Disease Data Interactium—is developing an Interactium®, a patient-centred real-world data platform for neuromuscular diseases (NMD).

FSHD Europe is supporting this work as a subcontractor through TREAT-NMD, alongside Work Package 9 lead FSHD Society. This work is aligned with Project Mercury in addressing an important challenge: as promising FSHD therapies move closer to approval, stronger health-economic evidence and robust disease-progression models will be essential to support timely reimbursement decisions and patient access.

FSHD Europe joins a global call for stronger population-level research to better understand mortality and life expectancy across FSHD and support the community.

Satellos Bioscience, a biotechnology company in Canada, has received clearance from the US Food and Drug Administration (FDA) to begin a Phase 2 clinical trial of its investigational medicine, forazapadin, in adults living with facioscapulohumeral muscular dystrophy (FSHD). The company has also announced a funding partnership with the FSHD Canada Foundation that could provide up to US$5 million to support the programme.
The trial is expected to begin later in 2026 and will evaluate the investigational therapy in adults living with FSHD.

FDA clearance allows Dyne Therapeutics to begin the first Phase 1 clinical trial of DYNE-302, an investigational therapy targeting DUX4 in FSHD.

16 July 2026 The global FSHD community came together in Chicago for the FSHD International Research Congress (IRC) 2026, FSHD Connect, and the Project Mercury Global Task Force meeting – a week filled with scientific progress, collaboration, and renewed commitment to...

15 July 2026. On 9 July 2026, members of the Roche Patient Partnership Team and European representatives of the FSHD Patient Advocacy Group met to discuss the results of the Phase 2 MANOEUVRE study of the investigational therapy emugrobart for FSHD. Roche has worked...

Novartis has announced encouraging results from the Phase I/II FORTITUDE clinical trial of delpacibart braxlosiran (del-brax), an investigational treatment for FSHD. The study met its primary biomarker endpoint, marking an important step forward in the development of potential disease-modifying treatments for FSHD.

Encouraging early results from the EPI-321 clinical trial for FSHD

Recent news from the FSHD research pipeline has brought cautious optimism to our FSHD community. Epicrispr Biotechnologies has announced encouraging early findings from its ongoing Phase 1/2 clinical trial of EPI-321, an investigational therapy designed to target the root cause of FSHD.

Italy’s FSHD community gathers in Rome on World FSHD Day
On 20 June 2026, FSHD Italia APS will host the XII National FSHD Conference in Rome — on World FSHD Day itself — bringing together patients, caregivers, clinicians and researchers for a full day of science, advocacy and community. The event takes place at UNA Hotels Decò (Sala Campidoglio), is free of charge and available in live streaming, with prior registration.
The conference is organised in collaboration with UILDM and the FSHD Group of AIM (Italian Association of Myology).

Today, March 19th, 2026, FSHD Europe announced its collaboration with TREAT-NMD and the PaLaDIn consortium to develop an FSHD disease model for health technology assessment (HTA), FSHD patient journey and patient advocacy toolkits. FSHD Europe strengthens its capacity...