TreatSMA has some exciting news to share about the new experimental drug being developed by Roche.

All the 24 young babies with SMA type 1 who since late 2016 joined the Firefish part I clinical trial continue to breathe and feed independently, the oldest of them being almost 2 years old now. No drug-related serious adverse events were observed that would result in any patient leaving any of the RG trials. What is most exciting, early data from the ongoing clinical trials indicated that RG7916 increased SMN protein level up to 6.5-fold when taken orally at a standard dose!

Deficiency of the SMN protein, caused by a genetic mutation, is the direct cause of the degeneration of motor neurons in those with spinal muscular atrophy. RG7916 works by modifying the way the SMN2 gene works so as it starts to produce the needed protein. The experimental compound is being developed by a consortium composed of SMA Foundation, PTC Therapeutics and a Swiss pharmaceutical giant F. Hoffmann–La Roche.

TreatSMA has been advocating for the clinical trials of RG7916 to start in the UK at the earliest.