MDL-101, for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), has received Rare Pediatric Disease (RPD) Designation from the U.S. Food and Drug Administration (FDA), and was featured on Cure CMD and LAMA2 Europe, respectively, as an achievement of Modalis Therapeutics.
In the article, it is mentioned that Modalis has been trying to develop innovative therapeutics for many years and has now achieved one significant milestone.
Article Title: One Step Closer to LAMA2-RD Clinical Trials: Modalis Therapeutics
About Cure CMD
Cure CMD was founded in 2008 by three parents whose children have congenital muscular dystrophy. Through close collaboration with researchers, clinicians, industry, advocates, and families, Cure CMD have made an incredible impact in the lives of those living with CMD. Cure CMD provide $4 million in research grants, launch clinical trials, and support natural history studies, and host scientific and family conferences etc.
About LAMA2 Europe
LAMA2 Europe is a non-profit organization organized and managed by the European LAMA2-CMD patient organizations (Netherlands, Spain, and France). The mission of LAMA2 Europe is to accelerate the development of treatments for LAMA2-CMD and to support patients and their families by providing relevant information on international research activities related to LAMA2-CMD, and by collaborating and connecting patients and their families with clinicians, researchers, and members of the biotech industry.