Thousands of children with severe developmental disorders have benefited from more targeted treatments and support with genetic insights from the large-scale Deciphering Developmental Disorders (DDD) ...
A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...
Rare diseases are hard to diagnose and often require costly genetic testing and visits to specialists. The testing can be hard to access, especially for people who live in rural areas. Researchers at ...
Diagnosing rare Mendelian disorders is a labor-intensive task, even for experienced geneticists. Investigators at Baylor College of Medicine are trying to make the process more efficient using ...
Genetic results from the Deciphering Developmental Disorders (DDD) study have enabled thousands of children with severe developmental disorders to receive better treatment. Thousands of children with ...
CINCINNATI, Nov. 17, 2025 /PRNewswire/ -- Cincinnati Children's today celebrated the opening of a new state-of-art facility for its Applied Gene and Cell Therapy Center, which will enable the health ...
For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation. The baby ...
Scientists have pinpointed genetic changes that can leave children born with little to no immune defence against infection. In a new study of 11 affected individuals, researchers from Newcastle ...
University of Otago – Ōtākou Whakaihu Waka has led international research uncovering a new genetic cause for a rare developmental disorder that profoundly impacts brain growth and function in children ...
A long-established drug with a diverse medical history is drawing new attention for its potential role in treating an ...
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