Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.
Haiyan ZhouJinhong MengAlberto MalerbaFrancesco CatapanoPalittiya SintusekSusan JarminLucy FengNgoc Lu-NguyenLianwen SunVirginie MariotJulie DumonceauxJennifer E MorganPaul GissenGeorge DicksonFrancesco MuntoniPublished in: Journal of cachexia, sarcopenia and muscle (2020)
These data suggest that myostatin inhibition, in addition to the well-known effect on muscle mass, can also positively influence the sensory neural circuits that may enhance motor neurons function. While the availability of the antisense drug Spinraza for SMA and other SMN-enhancing therapies has provided unprecedented improvement in SMA patients, there are still unmet needs in these patients. Our study provides further rationale for considering myostatin inhibitors as a therapeutic intervention in SMA patients, in combination with SMN-restoring drugs.
Keyphrases
- end stage renal disease
- ejection fraction
- newly diagnosed
- chronic kidney disease
- randomized controlled trial
- prognostic factors
- peritoneal dialysis
- emergency department
- skeletal muscle
- type diabetes
- stem cells
- adipose tissue
- mesenchymal stem cells
- patient reported outcomes
- insulin resistance
- metabolic syndrome
- weight loss
- deep learning
- big data
- replacement therapy