Joana Fernandes,  —

Interim results from the Phase 1/2 trial clinical trial investigating CAP-1002 as a treatment for heart disease associated with Duchenne muscular dystrophy (DMD) are being presented today at the 2017 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference taking place in Chicago. Linda Marbán, president and CEO of Capricor Therapeutics, is talking about the company’s plans…

The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California. The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five…

Levels of a protein called creatine kinase (CK) are a valuable biomarker for advanced Duchenne muscular dystrophy (DMD), but now researchers say it may also be a strong predictor of patients’ lung function. The study, “Correlation of Serum Creatine Kinase Level With Pulmonary Function in Duchenne Muscular Dystrophy,” was…

Increasing the levels of a muscle-inflammation-reducing hormone called adiponectin could help reverse the progression of Duchenne muscular dystrophy (DMD), a study suggests. The article in the American Journal of Pathology was titled “Potential Therapeutic Action of Adiponectin in Duchenne Muscular Dystrophy.” Adiponectin can reduce acute and chronic muscle inflammation. Previous…

Solid Biosciences’ SGT-001 improved muscle activity in dogs with Duchenne muscular dystrophy (DMD), according to research the company presented at a Washington conference. Another finding of the two preclinical-trial studies was that SGT-001 increased the expression of microdystrophin, a form of the protein dystrophin found in muscle. Too little of…

PerkinElmer has developed an improved screening method to test whether newborns may have Duchenne muscular dystrophy (DMD). The test detects the levels of a specific form of a protein called creatine kinase (CK), a disease biomarker, using dried blood spots from a baby’s blood sample. A report on these findings, “Characterization…

The journal Orphanet Journal of Rare Diseases has published a review highlighting the prevalence of Duchenne muscular dystrophy (DMD) as well as the disease’s costs and treatment patterns in North America and Europe.   The analysis, “The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…

CAP-1002, developed by Capricor Therapeutics, safely and effectively treats cardiac anomalies associated with Duchenne muscular dystrophy (DMD). So says a clinical study whose preliminary results formed the basis of an April 28 webinar jointly hosted by Capricor and the nonprofit groups Coalition Duchenne and Parent Project Muscular Dystrophy. DMD, which occurs in…

Resolaris, a protein therapy developed for rare muscle diseases by aTyr Pharma, improved muscle strength and quality of life in a small study of patients with early onset facioscapulohumeral muscular dystrophy (FSHD). These findings, as well as others regarding the effect of Resolaris in patients with limb girdle muscular…

Researchers from the National Institutes of Health have gained valuable insight into how cells in the heart and other muscles protect themselves from sudden plunges in energy production. Their findings shed light on how energy shortages affect patients with muscular disorders such as muscular dystrophy. And they may pave…