The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, ...
Apr 6, 2021
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A team from the Universitat Politècnica de València (UPV) and the CIBER Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN) has designed and tested, at a preclinical level, a new biomaterial for the treatment and ...
Mar 31, 2021
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New research gives hope medics might eventually be able to earlier diagnose and better treat a devastating and lethal muscle wasting disease.
Feb 18, 2021
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The Canadian Neuromuscular Disease Registry (CNDR) was launched in 2010 to increase efficient patient access to cutting-edge research and clinical trials, to increase understanding of the natural history and epidemiology ...
Feb 2, 2021
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An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.
Jan 28, 2021
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Cell-derived exosomes are effective in treating disease when mixed with the dominant protein in breast milk and given orally, a new Smidt Heart Institute study of laboratory mice shows. The findings, published in the peer-reviewed ...
Jan 12, 2021
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Trinity researchers are studying how the brain re-wires itself in neurological disease. The team is building treatments for today's more common global conditions like motor neurone disease (MND/ALS) and spinal muscular ...
Nov 17, 2020
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Muscular dystrophy, a condition that affects the body's muscles, comes with progressive muscle weakness and prolonged periods of muscle tightness. An estimated 975,000 to 3 million people worldwide have been diagnosed with ...
Nov 10, 2020
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A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests. The findings, published online ...
Nov 4, 2020
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A University of Alberta researcher's past work has led to a new drug being approved for use in the United States to treat patients suffering from Duchenne muscular dystrophy (DMD).
Oct 9, 2020
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