Attacking the flank: targeting new pathways in SBMA

Androgen withdrawal-based therapeutic strategies for spinal and bulbar muscular atrophy (SBMA) have shown limited benefit in clinical trials, and therapies for this disease still remain a considerable challenge. The finding that a class of migraine medications, the triptans, improve disease in a mouse model of SBMA suggests a new route for future investigations into SBMA therapies (pages 1531-1538).
Over 20 years have passed since the identification of the then-novel mutation that causes a rare motor neuron disease known as Kennedy's disease, or SBMA (1). That mutation, the expansion [...]