Research led by Professor Steve Wilton and Professor Sue Fletcher and licensed to Sarepta Therapeutics has delivered a second treatment for Duchenne muscular dystrophy, with the drug gaining accelerated approval by the U.S. ...
Jan 8, 2020
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Four-month-old Ainsley Cardente smiles, enjoys "helicopter" rides from her mom and protests when lunch is late. Perfectly ordinary, all of it. And her life is expected to remain that way thanks to a $2.1 million drug recently ...
Jan 6, 2020
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Swiss pharmaceutical company Novartis is preparing to give away 100 doses of the world's most expensive drug, which treats a rare childhood disorder, but its recipient selection process has drawn criticism.
Dec 20, 2019
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The causes of rare diseases could be uncovered using an approach created to identify genetic mutations that trigger a muscle-wasting condition, a study suggests.
Dec 18, 2019
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Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe ...
Dec 13, 2019
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Fresh insights into how nerves connect with muscles in the body could aid the development of therapies to treat neurodegenerative diseases.
Oct 30, 2019
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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 gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large ...
Oct 7, 2019
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A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD).
Sep 25, 2019
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A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
Sep 19, 2019
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