News

Becker MD Found in Boy with Epilepsy, Delayed Cognitive Skills

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…

Researchers Find 2 New Ways to Tackle RNA Diseases Such as MD

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…

UK Charity, Hope 4 Kidz, Helps to Remodel Home for Local Boy with DMD

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,…

Blood Pressure Medication Slows Heart Fibrosis, and Probably Heart Disease, in MD Patients

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…

TOR Inhibitor Eases Fibrosis and Improves Muscle Strength in Mice with MD Subtype

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…

Cognitive Decline Study in Late-onset DM1 Could Be Useful in Potential New Treatments

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.