Fresh insights into the protective seal that surrounds the DNA of our cells could help develop treatments for inherited muscle, brain, bone and skin disorders.
Feb 26, 2013
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New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. ...
Aug 9, 2013
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A natural history study has provided the first comprehensive clinical description of spinal muscular atrophy (SMA) within the Amish and Mennonite communities and correlates ancestral chromosome 5 haplotypes and SMN2 copy ...
Sep 6, 2018
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The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving ...
Jul 25, 2016
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(HealthDay)—In a head-to-head study of mineralocorticoid receptor antagonists, spironolactone was found to be noninferior to eplerenone for slowing the progression of heart damage in boys with Duchenne muscular dystrophy ...
Oct 28, 2019
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A puzzling question has lurked behind SMA (spinal muscular atrophy), the leading genetic cause of death in infants.
Feb 14, 2017
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A new study finds that muscle fibers in Duchenne muscular dystrophy (DMD) split during regeneration to such an extreme that the muscle is weakened beyond repair. The article is published ahead of print in the American Journal ...
Mar 1, 2018
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(HealthDay)—Drugs normally prescribed to treat erectile dysfunction in adult males may help boys who have a muscle disease called Duchenne muscular dystrophy, according to a new study.
May 7, 2014
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Neck floats marketed for babies to use in water can lead to serious injury or death, the U.S. Food and Drug Administration warned this week.
Jun 29, 2022
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Duchenne muscular dystrophy (DMD) is a muscular disease that shows symptoms in early childhood and causes progressive atrophy and eventual death. There is little in terms of treatment, partly because of poor understanding ...
Aug 20, 2015
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