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A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
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Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
Fragile X syndrome is a genetic condition associated with cognitive impairment, learning and behavioral challenges, and several physical features. Learn more about the disease here.
Fragile X is caused by mutations in the fragile X mental retardation 1 (FMR1) gene, located in the X chromosome. Learn more about what causes the disease here.
Symptoms differ in severity and occurrence among patients. Life expectancy for fragile X patients is generally normal. Learn more about the symptoms of the disease here.
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