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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A Virginia Tech team has identified a promising new therapeutic strategy for idiopathic pulmonary fibrosis (IPF), showing that blocking two proteins can halt and even reverse lung scarring in pre-clinical models.
Apr 28, 2026
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US health officials on Thursday greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss, a breakthrough that could pave the way for other such hearing impairment treatments.
Apr 24, 2026
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A new international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of Fudan University shows that a gene therapy for a rare form of genetic deafness successfully restored hearing in most ...
Apr 22, 2026
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University of Virginia School of Medicine researchers have used an advanced gene-editing technique to correct the underlying cause of a severe form of epilepsy in lab mice. This breakthrough could one day lead to new treatments ...
Apr 7, 2026
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Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration–approved ...
Mar 28, 2026
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Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single treatment is sufficient ...
Feb 26, 2026
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