Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic fluid, thus preventing damage that begins in utero.
May 14, 2025
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Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in utero treatment of SMA with the orally administered ...
Feb 20, 2025
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The U.S. Food and Drug Administration has approved a new drug application for a tablet version of Evrysdi (risdiplam) for people living with spinal muscular atrophy (SMA).
Feb 18, 2025
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Harvard stem cell biologists have pioneered a groundbreaking 3D organoid culture method for generating large numbers of adult skeletal muscle satellite cells, also known as muscle stem cells, in vitro.
Sep 12, 2024
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Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the ...
Jul 26, 2024
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In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or ...
Jun 25, 2024
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Spinal muscular atrophy (SMA) is a terrible disease in which a genetic mutation causes certain nerves responsible for sending signals to muscles to degenerate. This leads to muscles wasting away, and many patients have died ...
May 15, 2024
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Findings from a recent study in the Journal of Neuromuscular Diseases demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study's results add further support ...
Apr 15, 2024
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The lab of Yongchao C. Ma, Ph.D., at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago has discovered a fundamental biological mechanism that could lead to new treatments ...
Apr 5, 2024
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For the thousands of people globally affected by the disease myasthenia gravis (MG), everyday activities become a struggle, and in severe cases, the disease can be life-threatening.
Mar 21, 2024
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Spinal muscular atrophy (SMA) is an incurable autosomal recessive disease caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons, and resulting in death of neuronal cells in the anterior horn of spinal cord and subsequent system-wide muscle wasting (atrophy).
Spinal muscular atrophy manifests in various degrees of severity which all have in common general muscle wasting and mobility impairment. Other body systems may be affected as well, particularly in early-onset forms. Spinal muscular atrophy is the most common genetic cause of infant death.
Sometimes, the term spinal muscular atrophy is used to encompass other hereditary disorders that involve death of motor neurons in the anterior horn of spinal cord - see spinal muscular atrophies.
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