Targeted pharmacological treatment of autism spectrum disorders: fragile X and Rett syndrome


Today’s post is to refer the scientists among you to a very thorough paper looking at possible drug therapies for two specific variants of autism, Fragile X and Rett Syndrome.

Targeted pharmacological treatment of autism spectrum disorders: fragile X and Rett syndromes

  
These are single gene autisms and, as such, it is very much easier to study them than classic autism(s) or regressive autism(s).

We have already seen that much can be learnt from Fragile X and Retts.  What helps treat these disorders may give useful pointers to treat other types of autism and some therapies may be directly transferable, in some cases.









Note the use of baclofen, memantine, lovastatin, rapamycin, a PAK inhibitor, two potassium channel drugs, oxytocin, and even lithium.

Ganaxalone is a positive allosteric modulator of the GABAA receptor, probably affects the neurosteroid site.  It does not have the drawbacks of benzodiazepines.  I wonder whether it exhibits interesting effects at tiny doses? 

Tuning GABAa receptors
Treatment of Autism with low dose Phenytoin

Acamprosate appears to be neuro-protective, but the mechanism of action is unknown and controversial.  It is a drug a drug used for treating alcohol and benzodiazepine dependence.  A surprising number of off-label autism drugs are used for to treat substance abuse.

The paper is well worth a read for those who are heavily into the subject.






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