It may take an average of 11 years before people with Duchenne muscular dystrophy (DMD) lose upper body function and the ability to breathe on their own, according to a study by researchers of the HERCULES project, which set out to construct a natural history model of the…

People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using a wheelchair, a new study reports. The study, “Natural history of Becker muscular dystrophy: a multicenter study of 225 patients,” also provides new insights into the frequency of…

Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely seen only in adults — and type of mutation, a three-year study of BMD’s natural history shows. “We … provide evidence that decline/improvement in function would be hard to measure…

Researchers are planning to enroll a new group of patients in a natural history study to collect data about how Duchenne muscular dystrophy (DMD) progresses and affects a person’s life. Natural history studies don’t test an experimental therapy, but instead work to collect detailed information on how a disease…

A new natural history study that seeks to collect information about how Becker muscular dystrophy (BMD) progresses in the absence of treatment is now enrolling. The study is being led by the GRASP (General Resolution and Assessments Solving Phenotypes) consortium and Virginia Commonwealth University, its primary sponsor. Also collaborating…

Upper limb strength, function, and muscle fat fraction continuously worsen over time in Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 53 skipping, according to three-year data from a natural history study. Notably, this progression of upper limb involvement was assessed reliably with strength, function, and muscle…

The Myotonic Dystrophy Clinical Research Network (DMCRN) is asking adults with myotonic dystrophy type 1 (DM1) to participate in a large natural history study aimed at improving clinical trials by better understanding the full spectrum of the disorder. “Prior drug studies have been limited by a lack…

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also announced that children and adults with a confirmed LGMD2i genetic diagnosis are invited to join a natural history…

A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…

BBP-418, an oral treatment candidate for patients with limb-girdle muscular dystrophy type 2I (LGMD2I), is being developed by ML Bio Solutions, a new subsidiary of BridgeBio Pharma, the company has announced. BridgeBio intends to start a natural history study by…