A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hospital. Arthrogryposis, renal dysfunction and ...
Jun 19, 2026
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Muscle spasticity is a common and often debilitating consequence of spinal cord injury that can significantly affect mobility and quality of life. Spasticity is a condition in which muscles become abnormally stiff or tight ...
Jun 9, 2026
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An 8-month-old infant with severe genetic epilepsy has become the first patient in the world to receive an experimental gene replacement therapy designed to restore the function of the WWOX gene directly in the brain. The ...
Jun 8, 2026
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Fewer serious complications. Fewer hospitalizations and blood transfusions. Better growth and development. And a markedly lower risk of death from the complications of sickle cell anemia.
Jun 3, 2026
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MIT engineers have developed a noninvasive pacemaker that stimulates the heart using ultrasound. The design could one day provide a surgery-free alternative to traditional cardiac implants.
Jun 2, 2026
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Researchers have identified a key protein that may help failing hearts regain function, offering new insight into why some hearts recover while others do not. The discovery comes from studying patients treated with left ventricular ...
May 21, 2026
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For families of children born with alternating hemiplegia of childhood, the questions begin almost immediately: Will it get worse? How long do we have? What should we prepare for? The clearest answers yet are emerging and ...
May 17, 2026
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Researchers in the WashU Medicine Department of Ophthalmology & Visual Sciences have discovered key neuroprotective genes that could lead to the development of gene therapies to treat retinitis pigmentosa, an inherited form ...
May 11, 2026
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Thousands of times per year, a family's moment of joy turns to unexpected grief. A seemingly healthy infant stops smiling or making eye contact. Their limbs grow weak. The tiny child suffers seizures and breathing problems.
May 8, 2026
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An inherited form of blindness directly comparable to a common inherited optic nerve disease in humans has been discovered in rhesus macaques at the California National Primate Research Center at the University of California, ...
Apr 28, 2026
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