WORKSHOP AIM and OBJECTIVES
This EURO-NMD Workshop aims to bring together key stakeholders to improve the clinical management, equitable access, and sequencing of therapies for hereditary transthyretin amyloidosis (ATTRv) neuropathy. The core objectives include:
OBJECTIVE 1 – Clarify patient unmet needs and expectations
OBJECTIVE 2: propose a common diagnostic workflow
OBJECTIVE 3: propose minimal standards for treatment availability, delivery and follow-up
Besides the core objectives, we will explore strengthening early diagnostic strategies (including genetic and biochemical screening), reviewing the current landscape of disease-modifying and emerging therapies, and evaluating the real-world feasibility of treatment combinations and sequencing approaches. A central focus will be placed on discussing access across diverse health systems and on integrating patient and caregiver perspectives into care planning and research design.
WORKSHOP TARGET
The workshop is designed to convene all relevant stakeholders in the ATTRv field, including expert clinicians, basic and translational researchers, patient organisations, regulators, health technology assessors, and representatives from pharmaceutical and biotech companies developing or marketing therapies for ATTRv. By engaging all parties in a shared forum, the workshop seeks to identify practical short- and medium-term strategies to enhance care delivery and trial coordination across Europe.
WORKSHOP DISSEMINATION
A team of rapporteurs will summarise the key outcomes of the discussions. These findings will be synthesised into a consensus-driven summary paper, reviewed by all participants, and submitted for peer-reviewed publication within 6–12 months following the event. Where appropriate, working groups or follow-up initiatives will be launched to further develop proposals arising from the workshop.
WORKSHOP ATTENDANCE
The attendees will be mostly invited, but a select group of 10 to 20 attendees will be chosen from a list of registered candidates. You will be able to apply to participate by completing a form that will be available starting October 1, 2025. The scientific committee will select the participants based on the return received through the following form: click here to access the form.
All invited attendees will have transport and accommodation covered during the meeting and are expected to attend both days. Those registering for the meeting will have access to the meeting, breaks and work meals.
WORKSHOP PROGRAMME
WORKSHOP VENUE
Holiday Inn® Paris – Gare de Lyon Bastille
11 Rue De Lyon, Paris, 75012