Addex Therapeutics, CMTA Join Forces To Develop Potential Treatment For Rare Neurological Disorder

The Charcot–Marie–Tooth Association (CMTA), a philanthropic funder of CMT research worldwide, and Addex Therapeutics Ltd ADXN have collaborated to investigate a potential therapy for CMT type 1A (CMT1A).
Charcot-Marie-Tooth disease type 1A (CMT1A) is an inherited neurological disorder that affects the peripheral nerves.
People with this disease experience weakness and wasting (atrophy) of the muscles of the lower legs beginning in adolescence; later, they can also have hand weakness and sensory loss.
The primary goal of the collaboration is to evaluate the benefit of Addex's positive allosteric modulators (PAM) targeting the gamma-aminobutyric acid subtype B (GABAB) receptor in rodent models.
The GABAB receptor has previously been shown to be instrumental in controlling the Peripheral Myelin Protein-22 (PMP22) overexpression in a rat model of CMT1A.
Elevated PMP22 is closely associated with the disabling peripheral neuropathy that accompanies CMT1A.
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