In recent years, news have emerged from time to time that point to the potential benefit of some drugs for congenital muscular dystrophy patients. Although they do not hope to cure the disease, these drugs do seek to alleviate the harmful effects that a dysfunctional collagen matrix causes on muscle cells, slowly promoting their destruction. This is the case of alisporivir or givinostat which, like omigapil, have been frequently mentioned in publications and have been the object of both preclinical studies and clinical trials. In this article we summarize what is known about these products, their potential applicability in patients with collagen-VI muscular dystrophy and the current state of their clinical development.
On 30 de March de 20217 de May de 2021