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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Up to 45% of patients with the most common inherited neuromuscular disease could benefit from a new "cocktail" drug being developed at the University of Alberta, according to research published this week in the Proceedings ...
Feb 23, 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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Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, Ph.D., and colleagues have shown ...
Dec 23, 2021
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In a new study published in Brain Prof. Xi Chen from Nanjing University and Prof. Zhong Pei from Sun Yat-sen University report the therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs.
Dec 22, 2021
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Alexander disease is a progressive and rare neurological disorder with no cure or standard course of treatment. But a new study led by researchers at the University of Wisconsin–Madison involving a rat model of the disease ...
Nov 17, 2021
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Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. ...
Jun 29, 2021
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UNC School of Medicine scientists led a collaboration of researchers to demonstrate a potentially powerful new strategy for treating cystic fibrosis (CF) and potentially a wide range of other diseases. It involves small, ...
Jun 16, 2021
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Scientists from Nanjing University and University of Macau have discovered nano-scaled apoptotic bodies (ABs) as a new brain-targeting drug carrier, bringing new promise for the Parkinson's Disease as well as other brain ...
May 7, 2021
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Nagoya University researchers and colleagues in Japan have uncovered a molecular pathway that enhances chemotherapy resistance in some pancreatic cancer patients. Targeting an RNA to interrupt its activity could improve patient ...
Apr 21, 2021
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