Spain’s ongoing LAMA2-CMD natural history study

In 2022, a natural history study was initiated with LAMA2-CMD patients at Barcelona’s Vall d’Hebron Institute of Research.

Natural history studies follow a group of people over a period of time who have, or are at risk of developing, a specific disease or medical condition. They collect health information that helps to understand how the disease progresses and how to treat it. This information is also essential for designing and executing future clinical trials of new therapies.

Dr. Francina Munell is leading a natural history study with LAMA2-CMD patients at Vall d’Hebrón Hospital in Barcelona. The study has been ongoing since September 2022 after the Ethics Committee approved it in 2021. A total of 24 patients were enrolled, with a median age of 7.6 years (ranging from 1.6 to 54 years).

Patients have already undergone two yearly study visits. The next study visit will take place from October 4th to 6th, 2024, and will be conducted in person. During these days, patients will complete all study assessments, attend presentations from experts, and have the opportunity to interact and share their experiences with each other.