MDL-101, for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), and was featured on LAMA2 Europe as an achievement of Modalis Therapeutics.
Article Title:Modalis Therapeutics receives FDA Orphan Drug designation
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.