An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy (SMNRx)

Clinical Trial ID NCT01494701

PubWeight™ 16.73^‹?›

🔗 Visit the ClinicalTrials.gov page for NCT01494701

Top papers

Rank	Title	Journal	Year	PubWeight™‹?›
1	The spliceosome as a target of novel antitumour drugs.	Nat Rev Drug Discov	2012	2.51
2	Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy.	Neurology	2016	1.84
3	Antisense-based therapy for the treatment of spinal muscular atrophy.	J Cell Biol	2012	1.25
4	Direct intraventricular delivery of drugs to the rodent central nervous system.	J Vis Exp	2013	1.01
5	In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.	Mol Ther	2014	0.97
6	Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA.	Mol Ther Nucleic Acids	2012	0.95
7	Splicing therapy for neuromuscular disease.	Mol Cell Neurosci	2013	0.95
8	Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients.	PLoS One	2014	0.90
9	Advances in therapeutic development for spinal muscular atrophy.	Future Med Chem	2014	0.89
10	Development of therapeutic splice-switching oligonucleotides.	Hum Gene Ther	2014	0.88
11	Antisense therapy in neurology.	J Pers Med	2013	0.87
12	Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.	J Child Neurol	2016	0.87
13	Splice-switching antisense oligonucleotides as therapeutic drugs.	Nucleic Acids Res	2016	0.84
14	Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.	Nucleic Acid Ther	2014	0.83
15	Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery.	Nucleic Acid Ther	2014	0.81
16	Developing therapies for spinal muscular atrophy.	Ann N Y Acad Sci	2015	0.80