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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STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal ...
Aug 24, 2018
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A small molecule previously shown to enhance strength in injured or old laboratory mice does so by restoring lost connections between nerves and muscle fibers, Stanford Medicine researchers have found.
Oct 12, 2023
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Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic ...
May 8, 2018
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A new study from Cold Spring Harbor Laboratory (CSHL) reports surprising results that suggest that the devastating neuromuscular disease, spinal muscular atrophy (SMA), might not exclusively affect the motor neurons in the ...
Oct 5, 2011
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UCLA researchers have discovered the inner workings of a gene network that regulates the development of spinal motor neurons in the growing chicken and mouse embryo. The research also answers a long-standing question about ...
Feb 2, 2018
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Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment ...
Jul 14, 2022
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Spinal muscular atrophy (SMA) is a genetic disease that affects motor neurons in the spinal cord, resulting in muscle atrophy and widespread weakness that eventually impair swallowing and breathing. A new study in the Journal ...
Jul 16, 2018
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More than a billion people around the globe need assistive technology to go about everyday tasks independently. A new artificial intelligence (AI) wheelchair is taking assistive technology a step further and giving people ...
Jul 17, 2023
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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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