Table 2.

New therapeutic approaches and associated molecular pathways.

Medication/Compound	Molecular Mechanism	Clinical Trial Phases Completed	Reference
Curcumin	Decreases RANT and FMRpolyG production, reducing its accumulation.	Preclinical	[167]
	Selectively binds CGG RNA repeats potentially reducing RNA binding proteins sequestration and reducing CGG repeats-induced cell toxicity.
Cytidine 5′diphospho-choline (Citicoline)	Inhibition of phospholipase A2 (PLA2) leading to suppression of CGG repeats-induced cell toxicity.	Phase 2	[166,169]
Memantine	Noncompetitive antagonist of N-methyl-d-aspartate (NMDA) proposed to normalize premutation associated abnormal neuronal response to glutamate.	Phase 2	[161,170]
Piperine	Selectively binds CGG RNA repeats potentially reducing RNA binding proteins sequestration and reducing CGG repeats-induced cell toxicity.	Preclinical	[168]
Allopregnanolone	Positive allosteric modulator of GABA receptors.	Phase 2	[163,164,171,172]
	Reduction of caspase-3 protein expression leading to reduced apoptosis.
	Inhibition of mitochondrial permeability transition pore which is implicated in the intrinsic pathway of apoptosis.