Cookies on this website
We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy.

Journal article

McMillan HJ. et al, (2022), Expert Opin Biol Ther

Risdiplam: an investigational motor neuron-2 (SMN-2) splicing modifier for spinal muscular atrophy (SMA).

Journal article

Markati T. et al, (2022), Expert opinion on investigational drugs

Respiratory management of spinal muscular atrophy type 1 patients treated with Nusinersen.

Journal article

Menard J. et al, (2022), Pediatric pulmonology

Response of plasma microRNAs to nusinersen treatment in patients with SMA

Journal article

Zaharieva IT. et al, (2022), Annals of Clinical and Translational Neurology

Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy

Journal article

Fouarge E. et al, (2021), Orphanet Journal of Rare Diseases, 16

Crowdfunding for neuromuscular disease treatment: the ethical implications.

Journal article

Livingstone A. et al, (2021), Lancet Neurol, 20, 788 - 789

Load More