Current evidence for treatment with nusinersen for spinal muscular atrophy: a systematic review

B.06 Safety and efficacy of nusinersen in infants/children with spinal muscular atrophy (SMA): part 1 of the phase 2 EMBRACE study

(2018) PB Shieh et al.   CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Evidence in focus: Nusinersen use in spinal muscular atrophy

(2018) David Michelson et al.   NEUROLOGY

Efficacy and safety of nusinersen in infants with spinal muscular atrophy (SMA): Final results from the phase 3 ENDEAR study

(2017) R. Finkel et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Interim analysis of the phase 3 CHERISH study evaluating nusinersen in patients with later-onset spinal muscular atrophy (SMA): Primary and descriptive secondary endpoints

(2017) E. Mercuri et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Development of gene therapies—lessons from nusinersen

(2017) L Xu et al.   GENE THERAPY

How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy

(2017) N N Singh et al.   GENE THERAPY

The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

(2017) V Christie-Brown et al.   GENE THERAPY

Cure SMA and our patient community celebrate the first approved drug for SMA

(2017) J Glascock et al.   GENE THERAPY

Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy

(2017) S Jablonka et al.   GENE THERAPY

New treatments for serious conditions: ethical implications

(2017) N M P King et al.   GENE THERAPY

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape

(2017) Matthew J.A. Wood et al.   HUMAN MOLECULAR GENETICS

FDA-Approved Oligonucleotide Therapies in 2017

(2017) Cy A. Stein et al.   MOLECULAR THERAPY

Revised upper limb module for spinal muscular atrophy: Development of a new module

(2017) Elena S. Mazzone et al.   MUSCLE & NERVE

Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological Exam-Part 2: Experience from a nusinersen clinical study

(2017) Kathie M. Bishop et al.   MUSCLE & NERVE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Observational study of spinal muscular atrophy type I and implications for clinical trials

(2014) R. S. Finkel et al.   NEUROLOGY

The effectiveness of allied health care in patients with ataxia: a systematic review

(2013) Ella M. R. Fonteyn et al.   JOURNAL OF NEUROLOGY

Level of evidence reviews: Three years of progress

(2012) E. J. Ashman et al.   NEUROLOGY

Validation of the Expanded Hammersmith Functional Motor Scale in Spinal Muscular Atrophy Type II and III

(2011) Allan M. Glanzman et al.   JOURNAL OF CHILD NEUROLOGY

Spinal muscular atrophy type 1

(2011) Tara J. Lemoine et al.   Pediatric Critical Care Medicine