New Multi-Center Trial of Valproic Acid and Carnitine in Infants with Type I Spinal Muscular Atrophy.
Families of SMA is excited to announce a new clinical trial designed to evaluate the combination of Valproic Acid (VPA) and L-Carnitine for the treatment of SMA in infants with Type I SMA, called Carni-Val Type I.
Specifically, this trial will assess the safety of VPA and L-Carnitine in infants and develop improved methods to assess the strength and motor abilities of severely affected infants. The study is being fully funded by Families of SMA. Please see below for the formal trial announcement.
With new scientific discoveries and continued financial support, our community will see additional growth in the SMA drug pipeline and the need for more clinical trials. In addition to testing the safety of VPA and L-Carnitine in Type I infants, this trial will allow us to prepare for future clinical trials with novel SMA drugs by tailoring clinical trial protocols to SMA type.
