Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved in Europe—for example, ...
Aug 2, 2022
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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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In 2016, Spinraza became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death. The drug ...
Jul 13, 2022
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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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New research published in Developmental Medicine & Child Neurology indicates that early identification and treatment of patients with spinal muscular atrophy (SMA)—a genetic disorder characterized by weakness and wasting ...
Jun 8, 2022
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The stretches of DNA that differ from person to person, called variants, are a major part of what makes us unique, but they can also put us at greater risk of disease. Although we can currently spell out between 80% and 90% ...
Feb 7, 2022
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The World Health Organization projects that starting in 2030, over a million people will die each year from liver cancer. Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer, former postdoc Wai Kit Ma, and Dillon ...
Feb 3, 2022
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Researchers at Children's Hospital of Philadelphia (CHOP) successfully developed and validated a new outcome measure to monitor disease severity and progression in patients with impaired skeletal muscle function caused by ...
Sep 1, 2021
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Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
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Until recently, babies and children with spinal muscular atrophy (SMA) lacked any type of treatment capable of stopping the neuromuscular disease from progressing. In SMA, damaged motor neurons in the spinal cord fail to ...
Aug 20, 2021
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