Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...

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What Is Duchenne Muscular Dystrophy ... For the small number of DMD patients with a gene mutation that can be treated by skipping exon 45 (a certain section of genetic code), doctors can ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... DMD patients with DMD mutations amenable to exon 44 skipping ...

To be able to determine if mutations in specific domains of CAPN3 ... we included five clinically matched patients with Becker muscular dystrophy (BMD) (age 31 ± 16 years) and five patients ...

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

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 ...