People with certain mutations in the gene coding for dysferlin develop muscular dystrophy—a group of muscle wasting diseases that affect thousands around the world. Professor Simone Spuler and ...

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 ...

used a technique called antisense therapy to treat a mouse version of Duchenne muscular dystrophy. Small fragments of genetic code were used to skirt round the mutations in the dystrophin gene ...

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 ...

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 ...

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

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 ...

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 ...

Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically ...

The study evaluated ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD ... a rare and incurable pediatric disease caused by mutations in the gene encoding dystrophin, a protein ...