Patricia Inácio, PhD, science writer —

In a new study entitled “Under-recognition of Low Blood Pressure Readings in Patients with Duchenne Muscular Dystrophy,” researchers determined for the first time how blood pressure is affected in patients with duchenne muscular dystrophy. They report a significant portion of DMD patients exhibit low blood pressure, and highlight the need for clinicians…

In a recent study entitled “Progression of cardiac involvement in patients with limb-girdle type 2 and Becker muscular dystrophies: A 9-year follow-up study,” researchers describe a higher risk for cardiac dysfunction in patients suffering from Becker muscular dystrophy and limb-girdle muscular dystrophy, particularly subtypes 2I and 2E.

A new pilot clinical trial on Duchenne Muscular Dystrophy, entitled “Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Muscle Degeneration in Duchenne Muscular Dystrophy,” is now enrolling participants. The trial is a prospective observational study to test the capacity of a new ultrasound-based imaging…

A new study entitled “Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers” shows a new class of antisense oligonucleotides – tcDNA- antisense oligonucleotides – are highly efficient at rescuing dystrophin levels and improving symptoms in a mouse model of Duchenne muscular dystrophy.

A new study entitled “Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9” reports an iPSC-based gene therapy that can correct the genetic defect underlying Duchenne Muscular Dystrophy. The study was published in the journal…

Sarepta Therapeutics, Inc. recently announced a new study to confirm the dosages of their lead drug eteplirsen as a new therapeutic for treating Duchenne muscular dystrophy-affected patients with a baseline 6-minute walk test score. Duchenne muscular dystrophy (DMD) is a form of muscular dystrophy that is caused by a mutation of the Dystrophin gene,…

A team of researchers recently presented their latest results showing how injecting cardiac stem cells in mouse models of Duchenne muscular dystrophy can reverse damage to the heart. These findings carry a new therapeutic promise to patients with muscular waisting disorders. The study was presented in the American Heart…

A study entitled “Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients” describes the positive results of using a recombinant adeno-associated virus vector to restore dystrophin levels in dogs affected with Duchenne muscular dystrophy.

A new study entitled “Identifying diagnostic DNA methylation profiles for facioscapulohumeral muscular dystrophy in blood and saliva using bisulfite sequencing” published in the Clinical Epigenetics journal designed an assay that identifies the epigenetic signature for both types of Facioscapulohumeral muscular dystrophy from a multiple collections of…

The results from Milo Biotechnology’s Follistatin Gene Therapy were published in the Molecular Therapy journal showing therapy efficacy in increasing muscle strength and function in Becker’s muscular dystrophy patients. The study is entitled “A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.” The study followed…