Why is metformin used for Fragile X?
It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities.
Is Fragile X considered a disability?
Fragile X syndrome is characterized by moderate intellectual disability in affected males and mild intellectual disability in affected females. The physical features in affected males are variable and may not be obvious until puberty.
What is Fragile X life expectancy?
None of the symptoms of fragile X syndrome are life-threatening, so the life expectancy for someone with fragile X syndrome is the same as for the average person.
Can gene therapy cure Fragile X?
Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and monogenic autism. Individuals with FXS cannot live independent lives, and there is currently no treatment or cure available.
How does metformin XR work?
It works by decreasing the glucose (blood sugar) production in the liver, decreasing glucose absorption in the intestines, and boosting insulin sensitivity. Metformin improves insulin sensitivity by increasing glucose uptake in the body’s tissues.
What do you know about gene therapy?
Gene therapy is a medical approach that treats or prevents disease by correcting the underlying genetic problem. Gene therapy techniques allow doctors to treat a disorder by altering a person’s genetic makeup instead of using drugs or surgery.
What diseases can be treated with gene therapy?
Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.