DRUGphase2small molecule
salbutamol
Brand names: Ventolin, Proventil
Mechanism
β2-adrenergic receptor agonist. Increases SMN2 full-length mRNA in SMA fibroblasts (~2-fold). Improves respiratory strength in SMA type II (PMID 40410501).
Approved indications
asthma
Related claims (16)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| gene expression | Salbutamol has been shown to increase full-length SMN2 mRNA and functional SMN protein levels in SMA patient-derived fibroblasts (demonstrated in vitro). | 65% | 40410501 |
| motor function | Salbutamol treatment shows improvement in motor function in most SMA patients. | 59% | 30593463 |
| motor function | Salbutamol possesses the potential to improve motor function in patients with SMA. | 59% | 40410501 |
| drug efficacy | Salbutamol increases gem formation (a marker for SMN protein in the nucleus) in SMA patient fibroblasts in a dose-dependent manner. | 59% | 25514431 |
| drug efficacy | Salbutamol treatment significantly increases SMN2 full-length transcript levels in peripheral blood of SMA patients. | 59% | 30593463 |
| drug target | Salbutamol is a beta2-adrenergic agonist that modulates SMN2 levels. | 59% | 30593463 |
| pathway membership | The potential mechanism of salbutamol in treating SMA patients may involve enhancing SMN2 transcript expression via cAMP regulation and increasing SMN protein levels by inhibiting ubiquitin-mediated SMN degradation through the β2 adrenergic... | 59% | 40410501 |
| splicing event | Salbutamol elevated the levels of peripheral SMN2 full-length transcripts, with statistical significance observed at 6 months (MD = 25.13, 95% CI 16.12-34.13, P < 0.00001) and sustained through to 12 months (clinical trial result). | 56% | 40410501 |
| safety | Salbutamol seemed safe and was well tolerated without serious side effects. | 53% | 31201046 |
| safety | Short-term oral administration of salbutamol has good safety in later-onset SMA children (clinical trial result). | 53% | 38105669 |
| drug efficacy | Short-term oral administration of salbutamol may improve motor and pulmonary functions in later-onset SMA children (clinical trial result). | 53% | 38105669 |
| drug efficacy | Salbutamol therapy had no effect on VTAp during QB (p>0.05) (observed in patient cohort). | 51% | 27820869 |
| drug efficacy | Long-term oral salbutamol showed benefits in respiratory function in children with SMA (observed in patient cohort). | 45% | 28668232 |
| drug efficacy | Oral administration of salbutamol might be helpful in the maintenance of motor function in patients with SMA type II. | 45% | 31201046 |
| drug efficacy | Long-term oral salbutamol appeared to increase the strength of the inspiratory muscles in a small cohort of SMA II patients (observed in patient cohort). | 45% | 28668232 |
| safety | Salbutamol is safe and well tolerated in SMA patients. | 39% | 30593463 |
Off-Target Findings (0)
No Boltz-2 / Chai-1 off-target panel claims recorded for this drug.