Research
The increased understanding of the molecular mechanisms of disease in FXS has led to the development of therapies targeting the affected pathways. Evidence from mouse models shows that mGluR5 antagonists (blockers) can rescue dendritic spine abnormalities and seizures, as well as cognitive and behavioral problems, and may show promise in the treatment of FXS. Two new drugs, AFQ-056 (mavoglurant) and dipraglurant, as well as the repurposed drug fenobam are currently undergoing human trials for the treatment of FXS. There is also early evidence for the efficacy of arbaclofen, a GABAB agonist, in improving social withdrawal in individuals with FXS and ASD.
In addition, there is evidence from mouse models that minocycline, an antibiotic used for the treatment of acne, rescues abnormalities of the dendrites. An open trial in humans has shown promising results, although there is currently no evidence from controlled trials to support its use.
Read more about this topic: Fragile X Syndrome
Famous quotes containing the word research:
“Men talk, but rarely about anything personal. Recent research on friendship ... has shown that male relationships are based on shared activities: men tend to do things together rather than simply be together.... Female friendships, particularly close friendships, are usually based on self-disclosure, or on talking about intimate aspects of their lives.”
—Bettina Arndt (20th century)
“The great question that has never been answered, and which I have not yet been able to answer, despite my thirty years of research into the feminine soul, is “What does a woman want?” [Was will das Weib?]”
—Sigmund Freud (1856–1939)
“... research is never completed ... Around the corner lurks another possibility of interview, another book to read, a courthouse to explore, a document to verify.”
—Catherine Drinker Bowen (1897–1973)