SMA Foundation

Spinal muscular atrophy program enters phase Ib/IIa trial in patients with SMA

Thursday, November 20, 2014

PTC Therapeutics’ joint development program in spinal muscular atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a phase Ib/IIa study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA and investigate the safety and tolerability of an investigational survival of motor neuron 2 (SMN2) gene splicing modifier (RG7800) over 12 weeks. The achievement of this milestone triggers a $10 million payment to PTC from Roche.

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PTC Therapeutics, the SMA Foundation and Roche collaborate

Friday, January 24, 2014

PTC Therapeutics, the SMA Foundation and Roche have announced that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.

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