Researchers have developed a one-time gene therapy that may help ease obesity and related metabolic problems in people with Prader-Willi syndrome (PWS), according to a study in a mouse model of the disease. A single injection of the therapy, which uses a harmless version of the adeno-associated virus (AAV)…
A new study uses a mobile app to track the experiences of caregivers for people with Prader-Willi syndrome (PWS), with the aim of giving researchers insight into day-to-day life that could inform treatment development. “Pragmatic measurement is an important key to drug development that can provide insight into the…
More than half of children with Prader-Willi syndrome (PWS) show signs of spontaneous puberty, but more than three-quarters will have hypogonadism (reduced function of the reproductive organs), according to a study in Australia. That means patients generally will not attain full sexual maturity without therapies to normalize hormone levels.
Prader-Will syndrome is a rare and complex genetic condition that results in a variety of physical and mental symptoms. Learn more about the disease here.
Prader-Willi is caused by genetic changes in a particular area of chromosome 15. Learn more about what causes the disease here.
Symptoms of Prader-Willi range from physical impairments to cognitive and behavioral issues. Learn more about the symptoms of the disease here.
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