Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps

T Gidaro, L Servais - Developmental Medicine & Child …, 2019 - Wiley Online Library
… the clinical benefit of nusinersen in subgroups of patients with SMA. Nusinersen represents
the first … En esta revisión ejemplificamos el beneficio clínico de Nusinersen en subgrupos de …

Nusinersen for the treatment of spinal muscular atrophy

CA Chiriboga - Expert review of neurotherapeutics, 2017 - Taylor & Francis
… Areas covered: This article reviews the results of the published open label nusinersen … yet
unpublished nusinersen–sham controlled trials, as well as the presymptomatic nusinersen trial …

Nusinersen: a treatment for spinal muscular atrophy

MK Claborn, DL Stevens, CK Walker… - Annals of …, 2019 - journals.sagepub.com
… and safety of nusinersen (Spinraza) in the treatment of spinal muscular atrophy (SMA). Data
… using the terms nusinersen, ISIS-SMN (Rx), and spinal muscular atrophy. Manufacturer …

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study

G Acsadi, TO Crawford, W Müller‐Felber… - Muscle & …, 2021 - Wiley Online Library
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease
characterized by progressive weakness and atrophy of the … Nusinersen is a synthetic antisense …

Nusinersen for spinal muscular atrophy

CD Wurster, AC Ludolph - Therapeutic advances in …, 2018 - journals.sagepub.com
… , nusinersen has to be applied every 4 months into the cerebrospinal fluid. In initial studies,
repeated lumbar … Considering that the treatment with nusinersen may become an integral part …

Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies

BT Darras, CA Chiriboga, ST Iannaccone… - Neurology, 2019 - AAN Enterprises
nusinersen improves motor function in children with later-onset SMA. Spinal muscular
atrophy (SMA) is a neuromuscular disorder characterized by progressive muscular atrophy and …

Nusinersen versus sham control in later-onset spinal muscular atrophy

E Mercuri, BT Darras, CA Chiriboga… - … England Journal of …, 2018 - Mass Medical Soc
… It has been developed for the treatment of spinal muscular atrophy (… Spinal muscular atrophy
(SMA) is an autosomal recessive neuromuscular disorder that is characterized by atrophy

FDA approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides

A Aartsma-Rus - Nucleic acid therapeutics, 2017 - liebertpub.com
Spinal muscular atrophy (SMA) is an autosomal recessively inherited disease, which is
characterized by progressive loss of motor neurons, leading to muscle atrophy … where nusinersen

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

DR Corey - Nature neuroscience, 2017 - nature.com
Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy.
Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as …

[HTML][HTML] Nusinersen versus sham control in infantile-onset spinal muscular atrophy

RS Finkel, E Mercuri, BT Darras… - … England Journal of …, 2017 - Mass Medical Soc
… We conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety
trial of nusinersen in infants with spinal muscular atrophy. The primary end points were a motor-…