Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.
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Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), will award a $600,000 grant to Nationwide Children's Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease.
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Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. Researchers at Nationwide Children's Hospital have shown that a gene delivery strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle - directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength.
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A potential vaccine for Alzheimer's disease also has been shown in mice to slow the weakening of muscles associated with inclusion body myositis, a disorder that affects the elderly.
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Researchers have cleared a safety hurdle in efforts to develop a gene therapy for a form of muscular dystrophy that disables patients by gradually weakening muscles near the hips and shoulders.
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A new UC Irvine study finds that lithium chloride, a drug used to treat bipolar disorder, can slow the development of inclusion body myositis, a skeletal muscle disease that affects the elderly.
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Researchers have discovered the first microRNAs - tiny bits of code that regulate gene activity - linked to each of 10 major degenerative muscular disorders, opening doors to new treatments and a better biological understanding of these debilitating, poorly understood, often untreatable diseases.
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LaShan Davis, featured this week on The Discovery Channel's “Mystery Diagnosis” spent three 10-hour days filming the segment and reliving the terrifying time she spent waiting for her “mystery disease” to be diagnosed.
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A trial of beta-interferon-1a in inclusion-body myositis (IBM) has failed to find benefit from the drug in this disease. Beta-interferons counteract some of the activities of the immune system, which some experts believe is misguided in IBM.
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