Work at Emcure Pharmaceuticals Ltd. and Peptris Technologies has led to the identification of new fused bicyclic heterocyclic derivatives acting as histone deacetylase (HDAC) inhibitors.
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).
The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
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.
Solve FSHD and Modalis Therapeutics Corp. have established a strategic collaboration to develop an innovative therapy for facioscapulohumeral muscular dystrophy (FSHD).
The U.S. FDA gave Sarepta Therapeutics Inc.’s rAAVrh74 viral vector, used in an investigational gene therapy for the treatment of limb-girdle muscular dystrophy, a step up, making it one of the first platforms to receive the agency’s platform technology designation.
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.
Work at Satellos Bioscience Inc. has led to the identification of new AP2-associated protein kinase 1 (AAK1) inhibitors reported to be useful for the treatment of muscular dystrophy.
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with progressive muscle degeneration. There is currently no approved treatment targeting the underlying cause of the disease.
