A new trial aimed at patients with type 3 SMA is being launched by Catalyst Pharmaceuticals for those living in both Italy and Serbia. The trial is designed to look at the efficacy of a drug called amifampridine.
Very little is known about this particular study and to the best of our knowledge the company hasn’t reached out to any patient advocacy groups. The trial is aimed at patients between the age of 6 and 50, who are able to walk up to 30 m independently and who are receiving no other treatment for SMA. More information can be found on the clinical trials website, but we question the benefit and what is going to be learnt from a trial that is scheduled to last just 8 weeks.