The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving ...
Jul 25, 2016
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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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Professor Megumu K. Saito and his colleagues have revealed that SMN, a protein involved in spinal muscular atrophy (SMA), promotes the functional maturation of mitochondria by regulating miRNA expression along with MYOD1 ...
Feb 17, 2023
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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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Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat ...
Jun 20, 2012
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Spinal muscular atrophy (SMA), the most common form of neurodegenerative disease in childhood, is caused by defects in the SMN1 gene. SMA patients potentially benefit from three recently approved disease-modifying therapies. ...
Nov 7, 2022
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There is now further evidence that a drug that is effective in treating the rare muscle-wasting disease spinal muscular atrophy (SMA) early in life may be associated with improvement in older children, according to a study ...
Apr 24, 2019
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There are numerous severe, difficult-to-treat monogenetic diseases that are caused by a defect in a single gene—also in the liver. These include the blood coagulation disorders hemophilia A or B or the metabolic disease ...
Oct 26, 2022
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Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, ...
May 10, 2012
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Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center ...
Jul 27, 2017
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