Orphanet Journal of Rare Diseases

Abstracts from the First European Meeting for ATTR Amyloidosis for Doctors and Patients

Paris, France. 02-03 November 2017

Orphanet Journal of Rare Diseases201712(Suppl 1):165

https://doi.org/10.1186/s13023-017-0710-5

Published: 2 November 2017

Introduction

David Adams1, Philip Hawkins2, Hartmut Schmidt3

1Department of Neurology, NNERF, Bicêtre Hospital, Assistance Publique - Hôpitaux de Paris, Paris-Sud University, Le Kremlin-Bicêtre, France; 2National Amyloidosis Centre, Division of Medicine, University College London, London, UK; 3University Hospital of Muenster, Muenster, Germany

ATTR amyloidosis comprises a group of rare multisystem diseases including non-hereditary wild type ATTR amyloidosis (also known as senile cardiac amyloidosis, senile systemic amyloidosis), Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FAC).

FAP was first described in the 1950s in Portugal presenting as autosomal dominant disease, whereas wild type ATTR amyloidosis was recognized in the 1980s as a non-hereditary form of restrictive cardiomyopathy in older patients.

ATTR amyloidoses are global diseases that are now being identified in most countries. They are progressive and life-threatening, and diagnosis is usually delayed.

The First European meeting for ATTR amyloidosis for doctors and patients (Fig. 1) will inaugurate a new era in relations between patients, between doctors, and between doctors and patients from many European countries with the ultimate aim of improving diagnosis, treatment and care of this serious disease.

Advocacy and Education experts from international institutions including EURORDIS (the Voice of Rare Disease Patients in Europe), ARC (Amyloidosis Research Centre), and the ISA (International Society of Amyloidosis) will be present.

Since it is the first event of this nature, we propose three interconnected meetings over the course of two days. On Day One, Patients and Doctors will each have their own meeting; on Day Two, Patients and Doctors will share a common session.

During the meeting for specialists, six keynote lectures and 59 abstracts will be presented, comprising 11 oral communications and 48 posters. Final data from two major phase 3 clinical trials for TTR-FAP will be presented.

Eleven national Patients’ Organizations will participate. Attendees will include specialists predominantly from Europe but also from USA, South America and Asia.