SMA Drug Study Seeks More Participants

A study of valproic acid and carnitine in types 2 and 3 spinal muscular atrophy (SMA) is seeking additional participants.

Laboratory experiments have suggested that valproic acid may increase the amount of full-length SMN (survival of motor neuron) protein in cells from SMA patients. A lack of full-length SMN is the underlying cause of SMA.

Carnitine has been added because valproic acid depletes supplies of this cellular compound.

See Valproic Acid and Carnitine in Patients With SMA: Phase 2 Study for details.