On the 17-19th of March 2023 doctors, researchers, patients and families gathered in Barcelona for the second international conference on LAMA2-CMD: Paving the Road to Therapy.
The goal of the conference was to exchange knowledge and research progress on LAMA2-CMD and establish fruitful collaborations between researchers, clinicians and patient representatives. More specifically, the conference aimed to address:
- Aligning patient registries and natural history studies and define biomarkers and guidelines which can be used in patient-care
- Defining diagnostic criteria and guidelines for alleviating symptoms and maintaining quality of life with a dissemination plan for Europe
- Listing available patient samples for research, sharing confidential research progress and establishing a LAMA2-MD therapy task-force
Over 40 experts (including clinicians, researchers and patients/parents/patient advocacy organizations) were present in person and over 108 particpants from 28 countries were also able to join the conference on-line. In addition to presenting scientific data, researchers and clinicians also participated in workshops alongisde patients and families to discuss questions and future collaborative efforts.
As a result of the conference, the international LAMA2-CMD community agreed to work towards achieving the following objectives:
- Connect national patient registries together.
- Align natural history studies.
- Estblish an inventory of relevant biomarkers.
- Develop guidelines for usage in regular care throughout the EU within the ERN-NMD, bridging the gap with the US
- Curate and distribute diagnositc criteria and standards of care.
- Involve patients in the overall clinical and reserch path.
- Form ‘umbrella’ patient organization to represent countries with no local patient organization.
- Establish a database of shared patietn samples for future, collaborative research.
- Actively collobarate as an international-task force on therapy development.
- Connect with other rare nueromuscular diseases representatives.
- Establish regular meetings to provide updates and promte regular scientific discussions.
The conference was organised by Maastricht University and the Dutch foundation Voor Sara, in cooperation with Lama 2-Europe. The 3-day conference was largely funded by a grant of the European Joint Programme on Rare Diseases.