To help alleviate the long-term adverse effects of glucocorticoids in patients with Duchenne muscular dystrophy (DMD), a research team at Sapienza University of Rome is investigating a new form of anti-inflammatory medication that acts on inflammatory pathways in the disease. By targeting a specific mediator of inflammation in DMD, the…
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Researchers at the Second University of Naples in Italy recently described the clinical characteristics of patients with a specific deletion in the dystrophin gene. The study was published in the journal Acta Myologica and is entitled “Clinical features of patients with dystrophinopathy sharing…
Stem cell therapy may be an interesting approach to treat Duchenne muscular dystrophy patients who also have Diamond-Blackfan anemia. A research team from the Department of Haematology & Bone Marrow Transplantation at Army Hospital in India conducted a single-patient study to see if allogeneic hematopoietic stem cell transplantation could work…
Researchers have been exploring the use of stem cell therapeutics for the treatment of severe conditions such as type 1 diabetes, Alzheimer’s disease and Muscular Dystrophy. There are several types of stem cells that scientists think may be used in different ways to develop treatments for muscular dystrophy. The main stem-cell-based approaches…
Researchers at Royal Victoria Infirmary and the Institute of Genetic Medicine in the United Kingdom recently conducted a review on the short stature and delayed puberty often seen in patients with Duchenne muscular dystrophy (DMD). The study was published in the journal Archives of Disease…
Fairfax, Virginia based eye tracking software developer LC Technologies, and screen protector and glare-elimination technology specialists NuShield, Inc. of Newtown, Pennsylvania ate teaming up to bring communication mobility to disabled patients who are unable to communicate via speech or hand motion. Eye tracking is the field of monitoring…
A scientist who helped develop a pioneering treatment recently expressed her frustration after the NHS refused funding of Translarna for the treatment of Duchenne Muscular Dystrophy. Translarna has recently become the first Duchenne Muscular Dystrophy targeting therapy to receive approval by the European Medicines Agency (EMA). The drug is available in many…
Researchers at the University of Oxford in the United Kingdom recently published in the journal Experimental Physiology a review on the recent progress in genetic based therapies for Duchenne muscular dystrophy (DMD). The study is entitled “Advances in genetic therapeutic strategies for Duchenne…
Myotonic muscular dystrophy type 1 is a chronic and slowly progressing genetic disease affecting many organs. It is the most frequent form of muscular dystrophy and, as the name implies, is characterized by wasting of the muscles throughout the body. Nevertheless, the genetic defect on the myotonic dystrophy protein kinase also…
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Catabasis Pharmaceuticals Begins MoveDMD Trial For The Treatment of Duchenne Muscular Dystrophy
Massachusetts based Catabasis Pharmaceuticals, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on their proprietary pathway pharmacology technology platform, recently announced that they have begun treating patients in their MoveDMD trial, a Phase 1/2 trial of CAT- 1004 for the treatment of Duchenne…
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