Effect of salbutamol on respiratory muscle strength in children with SMA: A pilot study

Khirani, Sonia; Dabaj, Ivana; Amaddeo, Alessandro; Olmo-Arroyo, Jorge; Tirolien, Stéphane; Coudert, Véronique; Estournet, B.; Fauroux, Brigitte; Quijano‐Roy, Susana

doi:10.1183/13993003.congress-2016.pa1220

Cited by 1 publication

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“…109 Salbutamol, a b2-adrenoreceptor agonist, modestly improves muscle strength in patients by increasing levels of SMN2 fulllength mRNA and protein 110 and possibly through stabilization of acetylcholine receptor clusters at the NMJ. 111 Although small clinical studies suggest that it could help maintaining motor function 64 or improve respiratory function 112 of SMA type 2 patients, large-scale, placebo-controlled studies are lacking.…”

Section: Bioenergeticsmentioning

confidence: 99%

Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine

Chaytow

Faller

Huang

et al. 2021

Cell Reports Medicine

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Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the most severe cases and when left untreated, leads to death within the first two years of life. Recent therapeutic advances have given hope to families and patients by compensating for the deficiency in survival motor neuron (SMN) protein via gene therapy or other genetic manipulation. However, it is now apparent that none of these therapies will cure SMA alone. In this review, we discuss the three currently licensed therapies for SMA, briefly highlighting their respective advantages and disadvantages, before considering alternative approaches to increasing SMN protein levels. We then explore recent preclinical research that is identifying and targeting dysregulated pathways secondary to, or independent of, SMN deficiency that may provide adjunctive opportunities for SMA. These additional therapies are likely to be key for the development of treatments that are effective across the lifespan of SMA patients.

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