Congenital muscular dystrophy type 1A (MDC1A)
Congenital muscular dystrophy type 1A (MDC1A) is an ultra-rare orphan disease and a very severe form of muscular dystrophy caused by mutations in the gene encoding laminin alpha2 chain, an extracellular matrix protein. As there is no cure for MDC1A, patients have a degraded quality of life and often die before reaching adolescence. Although progress is being made to develop treatments for some forms of muscular dystrophy, most notably for Duchenne Muscular Dystrophy, little or no progress has been made in addressing the unmet medical needs of MDC1A patients.
The aimed drug properties are improved quality of life resulting in a prolonged life span for these patients.
Merozyne Therapeutics´ drug development program offers a new and unique therapy opportunity to treat MDC1A patients. The program includes the development of New Chemical Entities and evaluation of these in relevant models.
The overall objective is to develop a pharmacological treatment for treat MDC1A.