Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It's an important hurdle to clear, ...
Nov 11, 2020
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(HealthDay)—The U.S. Food and Drug Administration has approved the first oral, at-home drug for spinal muscular atrophy (SMA), the agency announced Friday.
Aug 11, 2020
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The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in ...
Jul 2, 2020
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The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. ...
Jun 11, 2020
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The enzyme Gemin3 was identified as the molecular 'bridge' between genes whose mutation or disruption causes amyotrophic lateral sclerosis (ALS), according to a new study in Nature's Scientific Reports from scientists at ...
Jan 16, 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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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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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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