Sarepta Therapeutics, Inc., a biopharmaceutical company focused on the development of innovative RNA-based therapeutics, recently announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for the company’s eteplirsen. A NDA is the vehicle through which drug sponsors formally propose FDA to approve a new…
A study recently published in the journal Scientific Reports reported the development of a novel in vitro model of the early pathogenesis of Duchenne muscular dystrophy (DMD) using stem cells. The study is entitled “Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human…
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…
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…
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…
A study recently published in the journal Biochimica et Biophysica Acta (BBA) revealed new insights into the roles played by a specific metalloprotease in a mouse model of Duchenne muscular dystrophy (DMD). The study was led by researchers at Shinshu University School of Medicine and…
Researchers at Peking University in China recently revealed new insights on the involvement of mitochondria in muscle development and muscular dystrophy in animal models. The study was recently published in the journal PLoS One and is entitled “Remodeling of Mitochondrial Flashes in Muscular Development…
A study recently published in the Journal of Translational Medicine reviewed the link between taurine and skeletal muscle disorders including muscular dystrophy. The study entitled “Taurine: the appeal of a safe amino acid for skeletal muscle disorders” was conducted by researchers at Università…
A new study recently published in the journal ACS Nano revealed a new technique to trigger muscle cell differentiation from adult stem cells using a genetic approach. The study is entitled “Inducing Stem Cell Myogenesis Using NanoScript” and was conducted by researchers at…
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