| Study | Reason for exclusion |
|---|---|
| Abbara 2011 | Riluzole. Non‐randomised, open‐label study to assess pharmacokinetics of riluzole in people with SMA types II and III. |
| Brahe 2005 | Phenylbutyrate. Not randomised, not controlled. Pilot trial. Study on the effect of phenylbutyrate on human SMN expression in blood. |
| Brichta 2006 | Valproic acid. Not randomised, not controlled. Pilot trial. Study on the effect of valproic acid on human SMN expression in blood. |
| Chang 2002 | Hydroxyurea. Not randomised, not controlled. Pilot trial. Study on the effect of hydroxyurea on clinical manifestations and human SMN expression in blood. |
| Chiriboga 2016 | Nusinersen. Phase 1, 4 groups, dose‐escalating trial. Not randomised, not controlled. |
| Darbar 2011 | Valproic acid. Open‐label trial, not controlled. No placebo given. |
| EMOTAS 2014 | Pyridostigmine. Open‐label trial. Not randomised, not controlled. Study ongoing, but can be excluded. |
| Folkers 1995 | Coenzyme Q10. Not randomised, not controlled. Observational study that included 1 participant with SMA type III/IV. |
| Giovannetti 2016 | Salbutamol. No placebo given. |
| JEWELFISH 2017 | RG7916 or RO7034067. Open‐label trial, no placebo given. Study ongoing, but can be excluded. |
| JPRN‐JapicCTI‐163450 2016 | Sodium valproate. Open‐label trial, no placebo given. Study ongoing, but can be excluded. |
| Kato 2009 | Thyrotropin. Case report. |
| Khirani 2017 | Salbutamol. Not randomised, not controlled. Pilot trial. |
| Kinali 2002 | Albuterol. Not randomised, not controlled. Pilot trial. |
| Kissel 2011 | Valproic acid. Open‐label trial, not controlled. |
| Liang 2008 | Hydroxyurea. Not controlled. |
| Mercuri 2004 | Phenylbutyrate. Not randomised, not controlled. Pilot trial. |
| Merlini 2003 | Gabapentin. Not controlled (no placebo given, compared treatment with no treatment, not blinded). |
| Nascimento 2010 | Lamotrigine. Case series. Not controlled, not randomised. |
| NCT01703988 | Nusinersen. Open‐label study. Study completed; results pending. |
| NCT02052791 | Nusinersen. open‐label. Study completed; results pending. |
| NCT02876094 | Celecoxib. Open‐label, non‐randomised study. Primary outcome SMN protein level, no clinical scores or evaluation. Study ongoing, but can be excluded. |
| NCT03709784 | Nusinersen. Observational study in adults treated with nusinersen. Study ongoing, but can be excluded. |
| NPTUNE01 2007 | Sodium phenylbutyrate. Dose‐escalating study. Non‐randomised, not controlled. No placebo given. Trial terminated due to poor compliance with study drug administration. |
| OLEOS | Olesoxime. Not randomised, not controlled. No placebo given. Only people who participated in previous trials (TRO19622CLEQ11150‐1 or TRO19622CLEQ1275‐1) to be included. Study ongoing, but can be excluded. |
| Pane 2008 | Salbutamol. Not randomised, not controlled. Pilot trial. |
| Piepers 2011 | Valproic acid. Not controlled, not randomised. Case series. |
| Prufer de Queiroz Campos Araujo 2010 | Salbutamol. Pilot trial. Not controlled (no placebo given), not randomised. |
| Saito 2014 | Valproic acid. Case series. Not controlled (no placebo given), not randomised. |
| SHINE 2015 | Nusinersen. Open‐label, not randomised. No placebo or sham‐procedure given. Study ongoing, but can be excluded. |
| SMART01 | Valproic acid. Open‐label trial. Not randomised, not controlled (no placebo given). Study completed, no published data yet. |
| SMART03 | Valproic acid. Open‐label trial. Not randomised, not controlled (no placebo given). Study recruiting participants, but can be excluded. |
| Swoboda 2009 | Valproic acid and carnitine. Not controlled (no placebo given). Open‐label trial. |
| Tan 2011 | Salbutamol. Not randomised, not controlled. Case series. |
| Tsai 2007 | Valproic acid. Not randomised, not controlled. |
| Weihl 2006 | Valproic acid. Not randomised, not controlled. Retrospective study on people with SMA types III and IV. |
SMA: spinal muscular atrophy; SMN: survival motor neuron.