Roche’s lead compound to treat spinal muscular atrophy (SMA) is no more. Olesoxime is a neuroprotective agent that was purchased by Roche in 2015 from Tophos. To date, it has proven to not be effective in helping patients with either amyotrophic lateral sclerosis nor SMA.

As we have reported previously, the spinal muscular atrophy (SMA) community in Canada has been frustrated by the lack of support they have received by public drug plans that have severely restricted the use of Spinraza (nusinersen) to treat SMA patients.

If it were not for bad news, the spinal muscular atrophy (SMA) community in Canada would have no news at all. At least in the past couple of months.