Last updated: 4th January 2023

Researchers have tested other drugs in clinical trials. As a result they have then found them not suitable for treating SMA. These are some of them:

CK-2127107 / reldesemtiv (Cytokinetics)

CK-2127107 / reldesemtiv is a drug designed to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. It is being trialled for Amyotrophic lateral sclerosis (ALS) also known as Motor neurone disease (MND) but not now for SMA.

Amifampridine (Catalyst Pharmaceuticals)

Catalyst, a biopharmaceutical company focused on developing innovative therapies for people with rare debilitating neuromuscular and neurological diseases, was evaluating amifampridine phosphate, a neuronal potassium channel blocker, for the treatment of SMA.

LMI070 / Branaplam (Novartis)

Similar to RG7916, LMI070 was a small molecule drug that increases the amount of SMN protein made by the SMN2 gene. Development of Branaplam was discontinued in mid 2021.

Olesoxime (Trophos / Roche)

Olesoxime was a neuro-protective drug that supports the nervous system under stressful conditions. Development of Olesoxime was discontinued in mid 2018.