News
Markus Rüegg’s mission: Developing a gene therapy for LAMA2-CMD
This article is a translated and summarized version of an original German interview with Professor Markus Rüegg Hope for a…
The knowledge base
-
![]()
News
-
![]()
News
A look back on a conference to shape LAMA2 clinical care guidelines
We are delighted to reflect on the success of the ENMC conference, initiated by Voor Sara and Cure CMD. Together,…
-
![]()
News
Help shape the future of LAMA2-Related Dystrophy care guidelines
In January 2025, clinicians specialising in LAMA2-Congenital Muscular Dystrophy (LAMA2-CMD) will convene in The Netherlands to begin developing updated care…
-
![]()
News
Modalis Therapeutics receives FDA Orphan Drug designation
Modalis Therapeutics has been granted U.S. FDA’s grant of Orphan Drug Designation (ODD) for MDL-101, a cutting-edge treatment for congenital…
-
![]()
Research updates
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…
-
![]()
News
One step closer: FDA grants Rare Pediatric Disease designation to Modalis Therapeutics’ MDL-101 gene therapy
Modalis Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to…
The driving forces behind Lama2.com
Bekijk alle partners
