Could Droxidopa Become the First Symptomatic Therapy for Charcot-Marie-Tooth Disease? A Case Series (P12-11.027)

Charcot-Marie-Tooth disease (CMT) is a slowly progressing hereditary neuropathy with a diverse clinical presentation. The most classic phenotype includes distal lower extremity weakness with gait impairment progressing to upper extremity weakness combined with gradual loss of upper/lower extremity sensation. No pharmacological therapy has proven effective with management usually focusing on supportive care including physical rehabilitation, orthotics, and avoidance of neuropathy-exacerbating factors.