Patricia Inácio, PhD, science writer —

FibroGen, a biotechnology company with expertise in connective tissue growth factor (CTGF) and hypoxia-inducible factor (HIF) biology, recently announced enrolling the first two patients in its new, open-label, multicenter Phase 2 clinical trial to investigate the FG-3019 compound in Duchenne muscular dystrophy (DMD) patients.  The trial is currently recruiting participants and further…

In a new study entitled “Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype,” a team of researchers identified a single gene – Jagged1 – as the potential critical player capable of “rescuing” Duchenne muscular dystrophy phenotype in dogs that exhibit a complete absence of dystrophin. The study was published…

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.