News

A new 77-page GBI Research report entitled “Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy – Identifying and Commercializing First-in-Class Innovation” identifies and highlights a highly innovative and diverse pipeline of products for treating Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) Currently in the pipeline are 84…

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…

Pfizer is currently recruiting participants to enroll a phase 2 clinical trial that intends to assess the safety, efficacy and pharmacological parameters of its product, PF-06252616, in boys with Duchenne muscular dystrophy (DMD) who are able to walk (ambulatory). The clinical trial is registered under the identifier NCT02310763…

Messenger RNA (mRNA) and DNA are commonly thought of as the workhorse nucleic acids of a cell. Specialized proteins within a cell transcribe DNA strands into mRNA strands, which are then translated into proteins. Within a cell, there exist other types of nucleic acids that play important roles in maintaining…

A study recently published in the journal Skeletal Muscle revealed a new potential therapeutic approach to promote muscle regeneration in dysferlinopathy. The study is entitled “Upregulated IL-1β in dysferlin-deficient muscle attenuates regeneration by blunting the response to pro-inflammatory macrophages”, and was developed by researchers…

A recent study published in the renowned journal Nature revealed how energy is distributed in muscle cells to ensure their proper functioning. The study is entitled “Mitochondrial reticulum for cellular energy distribution in muscle” and was developed by researchers at the National Heart…

While groundbreaking discoveries rooted in the laboratory are making progress in treating children with Duchenne muscular dystrophy (DMD), acupuncture and Traditional Chinese Medicine (TCM) also hold potential to help alleviate symptoms and enhance muscle function. A team of researchers combined a number of alternative therapies along with drug therapy to…

A study recently published in the journal Nature Biotechnology revealed a new technique to generate muscle fibers in a laboratory setting, offering a better model to study muscular diseases including Duchenne muscular dystrophy (DMD). The study is entitled “Differentiation of pluripotent stem cells to…

New research out of Germany indicates that immune therapy might help treat Duchenne muscular dystrophy (DMD), a serious disease that causes muscle weakness. In a mouse model of the disease, the ability to run, as well as disease biomarkers, appeared to improve following antibody treatment, specifically a form of human…

Arizona-based Critical Path Institute (C-Path), a leading non-profit organization committed to accelerate and reduce developmental costs of medical products, recently announced it has formed its 10th consortium: The Duchenne Regulatory Sciences Consortium (D-RSC). Together with Parent Project Muscular Dystrophy (PPMD), the new consortium will utilize C-Path’s tested consensus science model to find a cure for…