Project Mercury – Global Taskforce Meeting 9th December 2023, Boston (USA)

At the beginning of December the Global Taskforce Meeting of Project Mercury took place in Boston in the US. Representing FSHD Europe Kees van der Graaf was one of the 20 participants attending the meeting in person, among them also Miriam Wagner-Long, the liaison and project manager for all European countries and as one of six virtual attendees Nicol Voermans, Chair of the FSHD European Trial Network (FSHD ETN) and Advisor of FSHD Europe was also present.

The three main points, that were discussed:

• Clinical Trial Readiness – Workplan was presented by David Allison, the CEO of Treat-NMD. The aim is an agreed minimum set of patient data which will meet the legally required data security. It is obvious that we need to get as many patients as possible in the different registries.
  The group also shared the outcome of the workshop in Banff. The Canadian Working Group (CaWG) will develop a template for the budgeting and organisation of the site. It will be essential that all sites have a clinician and MD trained physiotherapist who understands FSHD.

• Patient Access – Workplan was presented by Josie Godfrey. To get regulators and insurers on board, there must be an agreed FSHD disease model, an economic/cost illness model, and quality of life insights. It was mentioned that there will be a new EU legislation regarding access, which will replace all existing local and European legislation by 2025.
  Nicol Voermans presented results of a qualitative study amongst participants of the Losmapimod trials 2 and 3 in the Netherlands. In general, the results were positive. We learnt that it is important to spend more time on communicating the role of placebos.
  
• Sustainability – Workplan started with Canada’s insights on the importance of forming a strong relationship with MD organisations.
  Miriam Wagner-Long then presented the results of her landscape analysis.
  Brazil and Australia also shared their good progress.

There was also an update on PaLaDin (Horizon grant) which is aiming to strengthen patients voices through research and patient care. FSHD is considered a model to be replicated in other rare disease groups.
PaLaDin is the project’s name, and it stands for “Patient and Lifestyle Data Interactum”.

Further information and updates can be found on the project’s website:

Project Mercury – The Global Initiative to Speed the Delivery of Therapies for FSHD