Drug abates symptoms in two genetic models of autism
Drugs developed to treat fragile X syndrome may also work for autism because both disorders feature defects at neuronal junctions, suggests a paper published 12 January in Nature Neuroscience.
Drugs developed to treat fragile X syndrome may also work for autism because both disorders feature defects at neuronal junctions, suggests a paper published 12 January in Nature Neuroscience.
Mice missing the FMR1 gene only in star-shaped brain cells called astrocytes recapitulate key features of fragile X syndrome. Researchers presented the unpublished results today at the 2014 Society for Neuroscience annual meeting in Washington, D.C.
The protein missing in fragile X syndrome is necessary for mice to respond to the stimulant cocaine, according to a study published 7 May in Neuron.
There are no available medications for treating autism’s core symptoms, but there are several candidates in clinical trials. Jeremy Veenstra-VanderWeele describes the factors researchers must take into account when developing drugs for the disorder.
Following disappointing results from two clinical trials, the Swiss pharmaceutical company Novartis announced on 24 April that it will stop development of a drug candidate for fragile X syndrome.
Following the suspension in early May of two clinical trials of arbaclofen, a candidate drug for autism and fragile X syndrome, parents are appealing to the U.S. government and several pharmaceutical companies to continue testing the drug.
Researchers can use light to activate certain proteins that receive signals at the junctions between neurons and that are key targets for fragile X syndrome therapies, according to a study published in the April issue of Nature Neuroscience.
Researchers have added two new candidates to the arsenal of compounds that alleviate both the behavioral and molecular hallmarks of fragile X syndrome in mice that model the disorder. A third candidate, minocycline, improves some symptoms in children with the disorder.
A growing number of studies suggest connections between Alzheimer’s disease, fragile X syndrome and autism, which could point the way to potential treatments.
FMRP, the protein missing in fragile X syndrome, binds to the RNA sequences of 939 genes, 93 of which have been linked to autism, according to a study published 20 December in Nature.