CMT is one of the most commonly inherited neuromuscular diseases, with prevalence of approximately 1 in 2,500 persons. Clinical complains are mainly represented by muscle pain, sensation of fatigue and painful muscle cramps. No treatment of clinical symptoms is available yet. Previous treatment with high dosage of vitamin C failed to confirm a benefit in humans.
A clinical open trial has been performed in order to evaluate the efficacy of ultramicronized palmitoylethanolamide (PEA-um®). Twenty-two patients (7 male and 15 females) from four different CMT families were treated with PEA-um® at dosage of 1200 mg/day for 80 days (Normast ®, Epitech Group srl, Saccolongo, Italy). None of the patients had an add-on treatment for the clinical symptoms. Muscle pain, fatigue and muscle cramps were assessed at T0 (baseline), T1 (20th day) and T2 (80th day) using Visual Analogic Scale (VAS). Muscle strength, vibratory sensation and Motor/Sensory nerve Conduction velocities were also assessed with the same schedule. Mean values of VAS for muscle pain at T1decreased from 5.9 ± 2.1 to 3.9 ± 1.7 (p<0.0001), whereas VAS for fatigue decreased form 6.3 ± 2.4 to 3.4 ± 1.6 (p <0.0001). VAS score for painful cramps at T1diminished from 5.4 ± 1.2 to 3.8 ± 1.3 (p<0.0001). A further improvement of VAS scores for muscle pain, fatigue and painful cramps was observed at T2 evaluation.
These data strongly suggest that PEA-um® is effective in improving clinical symptoms of CMT neuropathy, albeit the obvious limitation of an open study.