ARTICLE | Cover Story
Selectively splicing SMN2
September 4, 2014 7:00 AM UTC
A team from PTC Therapeutics Inc., Roche and the Spinal Muscular Atrophy Foundation has generated selective, orally available, blood brain barrier-penetrant small molecule modulators of SMN2 splicing that are highly effective in mouse models of spinal muscular atrophy.1
The lead SMN2 (survival of motor neuron 2 centromeric) splice modulator discovered under the collaboration, RG7800, completed a Phase I trial in healthy volunteers this year. The partners are planning a trial to evaluate the candidate in patients with spinal muscular atrophy (SMA) but declined to disclose details...