News

CureDuchenne Ventures is investing in Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), a new specialized contract research organization. TRiNDS is hived off from the Cooperative International Neuromuscular Research Group (CINRG), an academic consortium that studies causes, pathogenesis and clinical outcomes of neuromuscular disorders, and conducts well-controlled clinical studies…

Dietary quercetin, an antioxidant belonging to group of plant pigments that gives many fruits and vegetables their color may partially protect muscles in Duchenne muscular dystrophy (MD), according to a study conducted on a mouse model of the disease and published in the scientific journal Plos One.

Patients with Duchenne muscular dystrophy and Becker muscular dystrophy treated with ACE inhibitors inhibitors (drugs that dilate blood vessels) had slower progression of myocardial fibrosis than those treated with usual care. The study with the findings, “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy: A Randomized Clinical Trial,” is…

Becker muscular dystrophy (BMD) was eventually diagnosed in a boy with epilepsy and dysgnosia (delayed cognitive development), but relatively age-normal running and walkingabilities, researchers report in what they called a rare case study. Later genetic testing revealed a duplicate mutation within the dystrophin gene, responsible for making a protein important…

Scientists have developed two new approaches to kill RNA molecules responsible for incurable diseases such as muscular dystrophy (MD),  according to a study. One is designing small molecules that destroy only disease-causing RNA. The other approach is using a disease-causing RNA as a catalyst to create a therapy that kills…

The U.K. charity Hope 4 Kidz has announced the successful completion of Matthew’s Milestone, a £75,000 (about $95,000) campaign to help a young boy with Duchenne’s muscular dystrophy (DMD) and his family. Matthew Brettel, an 11-year-old from Thorney Close, in northeast England, was diagnosed with DMD in 2013 and has been virtually wheelchair-bound since. Hope 4 Kidz,…

Treating Duchenne and Becker’s muscular dystrophy patients with angiotensin-converting enzyme (ACE) blocking drugs at early disease stages may slow the development of heart fibrosis and, as a consequence, offer patients a better prognosis. Those findings are in the study “Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy,” which was published…

Rapamycin administered to a mouse model of dystroglycanopathy, a particular form of muscular dystrophy, worked to reduce fibrosis and inflammation, researchers reported, while enhancing muscle strength. The study, “Four-week rapamycin treatment improves muscular dystrophy in a fukutin-deficient mouse model of dystroglycanopathy,” was published in the journal Skeletal Muscle. Eystrophin-glycoprotein…

A study describing the cognitive decline over time in adults with myotonic dystrophy type 1 (DM1) was published in the scientific journal Neuromuscular Disorders. The study, titled “Cognitive decline over time in adults with myotonic dystrophy type 1: A 9-year longitudinal study” assessed the cognitive ability of the participants over a period of nine years and is the longest study to date with the largest group of participants.

Researchers identified a group of cells called fibrocytes that may contribute to inflammation and fibrosis leading to muscle loss in Duchenne muscular dystrophy (DMD). The study, “Identification And Function Of Fibrocytes In Skeletal Muscle Injury Repair And Muscular Dystrophy,” was published in The Journal of Immunology. The muscle…