The 2nd International Conference on LAMA2-CMD

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:

  1. Aligning patient registries and natural history studies and define biomarkers and guidelines which can be used in patient-care
  2. Defining diagnostic criteria and guidelines for alleviating symptoms and maintaining quality of life with a dissemination plan for Europe
  3. 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:

  1. Connect national patient registries together.
  2. Align natural history studies.
  3. Estblish an inventory of relevant biomarkers.
  4. Develop guidelines for usage in regular care throughout the EU within the ERN-NMD, bridging the gap with the US
  5. Curate and distribute diagnositc criteria and standards of care.
  6. Involve patients in the overall clinical and reserch path.
  7. Form ‘umbrella’ patient organization to represent countries with no local patient organization.
  8. Establish a database of shared patietn samples for future, collaborative research.
  9. Actively collobarate as an international-task force on therapy development.
  10. Connect with other rare nueromuscular diseases representatives.
  11. 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.