Patricia Inácio, PhD,  science writer—

Debiopharm International SA, in partnership with Solid Biosciences, recently announced the initiation of pre-clinical animal studies of Alisporivir in the treatment of Duchenne Muscular Dystrophy (DMD). DMD is a severe form of muscular dystrophy caused by a mutation in the Dystrophin gene, located in the X chromosome, which results in the absence or abnormal production of…

In a new study entitled “Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells” a team of scientists developed an induced pluripotent stem cell model to study Duchenne muscular dystrophy and discover new therapeutic drugs. The study was published in the journal…

In a new study entitled “Differential Isoform Expression and Selective Muscle Involvement in Muscular Dystrophies”researchers performed a microarray analysis on human muscles to identify the molecular cause underlying the selectivity of muscles affected in muscular dystrophies. Their findings suggest that overexpression of mutated genes in specific muscles are…

In a new study entitled “Genome-wide Mechanosensitive MicroRNA (MechanomiR) Screen Uncovers Dysregulation of their Regulatory Networks in the mdm Mouse Model of Muscular Dystrophy” researchers performed a genome-wide study to identify whether mechanosensitive microRNAs are deregulated and contribute to muscular dystrophies pathogenesis. The study was published in…

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,…