The knowledge base

We are delighted to reflect on the success of the ENMC conference, initiated by Voor Sara and Cure CMD. Together,…

In January 2025, clinicians specialising in LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD) will convene in The Netherlands to begin developing updated care…

Modalis Therapeutics has been granted U.S. FDA’s grant of Orphan Drug Designation (ODD) for MDL-101, a cutting-edge treatment for congenital…

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

Modalis Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to…

We are thrilled to announce a new clinical workshop focused on establishing subtype-specific care recommendations for LAMA2-RD, and using this experience as a prototype to develop recommendations for…