Laminins are extracellular matrix (ECM) glycoproteins that preserve the structural and functional integrity of tissues. These heterotrimeric proteins, composed of one α, β and γ chain encoded by different genes, provide mechanical support, facilitate cell adhesion and maintain tissue organization and stability.

Arrakis Therapeutics Inc. has announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1).

Researchers have identified a potential therapeutic target for muscular dystrophies, a group of genetic disorders characterized by progressive muscle weakness and degeneration. The study reveals that inhibiting the microRNA (miRNA) known as miRNA-33 can significantly improve muscle regeneration and ameliorate the dystrophic phenotype in animal models.

Armatus Bio Inc.’s development of ARM-201, an AAV-delivered microRNA therapy for facioscapulohumeral muscular dystrophy (FSHD), has been boosted by a $3 million investment by Solve FSHD.

Recent work proposes a promising therapeutic approach for dysferlinopathies.