Category Archives: Muscular Dystrophy Treatment

Becker Muscular Dystrophy Treatment Market report contains key data about the market, emerging trends, product usage, motivating factors for customers and competitors. This is a detailed market research report that serves this purpose and gives the business a competitive advantage. The excellent market report evaluates the existing state of the market, market size and market share, revenue generated from the product sale, and essential changes required in the future products. Continue reading →

Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for Duchenne muscular dystrophy (DMD), its developer,Stealth BioTherapeutics, reported. Our new preclinical data increases our optimism that elamipretide may help address the unmet need for therapies in DMD, Reenie McCarthy, the CEO of Stealth, said in a press release. Mitochondria are often referred to as the powerhouses of the cell, because they are critical for energy generation Continue reading →

By Richard Robinson December 16, 2021 The Science Explained Article In Brief A new gene delivery vector and methodology called directed evolution enables more potent delivery of corrective genes to the muscles in mice and primate models, and holds promise for reducing the cost and risk of treatment for muscle disorders. A new gene delivery vector, building on over 20 years of basic virology research, holds promise for accelerating gene therapy research and reducing the cost and risk of treatment, according to a paper published September 16 in the journal Cell Continue reading →

Sheila Mikhail, CEO and Co-Founder of AskBio, was recently named EYs 2021 Entrepreneur Of The Year National Overall Award winner. The preeminent competitive awards recognize the unstoppable entrepreneurs transforming our world. Continue reading →

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Our theme ofOut Of Favor Health Care Stocksincludes healthcare and pharmaceutical stocks that are seeing a disconnect between their financial performance and recent stock price returns. Continue reading →

An international, multidisciplinary team of researchers from theTranslational Synthetic Biology Laboratoryat Pompeu Fabra University (Barcelona, Spain), led by Dr.Marc Gell, has published an article in the scientific journalNature Communicationsshowing the potential of Find Cut-and-Transfer (FiCAT) technology as a state-of-the-art tool forgene writingto develop advanced therapies that are safer and more effective in their future clinical application in patients withgenetic and oncological diseasesthat have few treatment options. Continue reading →

As children across the country count the sleeps until Santa Claus arrives, three Roscommon children with terminal conditions are hoping they will be able to move into their new home soon. Continue reading →

For Dr. Continue reading →

A Phase 1 clinical trial evaluating Edgewise Therapeutics experimental oral therapy EDG-5506 in healthy volunteers and men with Becker muscular dystrophy (BMD) has dosed the first BMD patients. The study (NCT04585464) is currently recruiting up to eight men, ages 18 to 55, with BMD at its single site in San Antonio, Texas; more information can be foundhere. Top-line data from these patients are expected by the end of the year Continue reading →