FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2790507197> ?p ?o ?g. }

• W2790507197 abstract "Objective: NURTURE (NCT02386553) is an ongoing phase 2, open-label, single-arm study, evaluating intrathecal nusinersen (12-mg equivalent dose) in infants with presymptomatic spinal muscular atrophy (SMA). Background: SMA is a neuromuscular disorder caused by a mutation in the survival motor neuron 1 ( SMN1 ) gene resulting in SMN protein deficiency. Nusinersen is an investigational antisense oligonucleotide drug that modifies SMN2 pre-mRNA splicing, promoting increased functional SMN protein production. Design/Methods: NURTURE is enrolling presymptomatic infants with genetically diagnosed SMA and evaluating their development from near birth to 2.5 years. The primary endpoint is time to death or respiratory intervention (tracheostomy/any ventilation support for ≥6 hours/day continuously for ≥7 days). Secondary endpoints include the proportion of infants developing clinical symptoms of SMA and achievement of motor milestones. Changes in compound muscle action potential (CMAP) and adverse events (AEs) also were assessed. Study enrollment started May 2015; interim results are reported. Results: As of 8 Jun 2016, 22 infants were screened; 17 were enrolled and dosed; 5 failed screening. Most (12/17) had 2 SMN2 copies, and most were aged ≤1 month at enrollment. All 17 infants remain enrolled; none have met the primary endpoints of respiratory intervention or death. Improvements in mean Hammersmith Infant Neurological Examination (HINE) motor milestones scores vs Baseline have been observed. Infants have generally gained weight over time consistent with normal development, and mean CMAP scores have mostly improved vs Baseline. There were no severe AEs reported, 5 infants experienced a serious AE. Three infants experienced AEs considered possibly related to study drug. No new safety concerns were identified. Conclusions: The NURTURE interim analysis shows that all the infants treated with nusinersen are alive and generally achieving new motor skills and appropriate age-related developmental gains. The efficacy and safety profile of nusinersen supports continued clinical development. Study Supported by: Biogen Disclosure: Dr. De Vivo has received personal compensation for activities with AveXis, Biogen, Roche, IONIS, Sarepta, Cytokinetics Pharmaceuticals, and the SMA Foundation. Dr. De Vivo has received research support from NIH, DOD, SMA Foundation,and Hope for Children Research Foundation. Dr. Hwu has nothing to disclose. Dr. Reyna has received personal compensation for activities with Biogen as an employee. Dr. Reyna holds stock and/or stock options in Biogen. Dr. Farwell has received personal compensation for activities with Biogen Idec as an employee. Dr. Farwell holds stock and/or stock options in Biogen Idec. Dr. Gheuens has received personal compensation for activities with Biogen as an employee. Dr. Sun has received personal compensation for activities with Biogen as an employee. Dr. Sun hold stock and/or stock options in Biogen. Dr. Zhong has received personal compensation for activities with Biogen Idec. Dr. Zhong holds stock and/or stock options in Biogen Idec. Dr. Su has received personal compensation for activities with IONIS Pharmaceuticals Inc. as an employee. Dr. Su holds stock and/or stock options in IONIS Pharmaceuticals. Dr. Schneider has received personal compensation for activities with IONIS Pharmaceuticals Inc. as an employee. Dr. Schneider holds stock and/or stock options in IONIS Pharmaceuticals. Dr. Bertini has received personal compensation for activities with AveXis, Biogen, Roche, Novartis, and Edison Pharmaceuticals as an advisor and/or consultant. Dr. Bertini has receives research support from Telethon and Italian Ministry of Health. Dr. NURTURE Study Investigators has nothing to disclose." @default.
• W2790507197 created "2018-03-29" @default.
• W2790507197 creator A5000779298 @default.
• W2790507197 creator A5015287299 @default.
• W2790507197 creator A5024035657 @default.
• W2790507197 creator A5040386348 @default.
• W2790507197 creator A5044252539 @default.
• W2790507197 creator A5049873494 @default.
• W2790507197 creator A5061100401 @default.
• W2790507197 creator A5061182813 @default.
• W2790507197 creator A5066921649 @default.
• W2790507197 creator A5080239287 @default.
• W2790507197 date "2017-04-18" @default.
• W2790507197 modified "2023-10-05" @default.
• W2790507197 title "Interim Efficacy and Safety Results from the Phase 2 NURTURE Study Evaluating Nusinersen in Presymptomatic Infants with Spinal Muscular Atrophy (S46.003)" @default.
• W2790507197 hasPublicationYear "2017" @default.
• W2790507197 type Work @default.
• W2790507197 sameAs 2790507197 @default.
• W2790507197 citedByCount "0" @default.
• W2790507197 crossrefType "journal-article" @default.
• W2790507197 hasAuthorship W2790507197A5000779298 @default.
• W2790507197 hasAuthorship W2790507197A5015287299 @default.
• W2790507197 hasAuthorship W2790507197A5024035657 @default.
• W2790507197 hasAuthorship W2790507197A5040386348 @default.
• W2790507197 hasAuthorship W2790507197A5044252539 @default.
• W2790507197 hasAuthorship W2790507197A5049873494 @default.
• W2790507197 hasAuthorship W2790507197A5061100401 @default.
• W2790507197 hasAuthorship W2790507197A5061182813 @default.
• W2790507197 hasAuthorship W2790507197A5066921649 @default.
• W2790507197 hasAuthorship W2790507197A5080239287 @default.
• W2790507197 hasConcept C114614502 @default.
• W2790507197 hasConcept C126322002 @default.
• W2790507197 hasConcept C161921814 @default.
• W2790507197 hasConcept C187212893 @default.
• W2790507197 hasConcept C197934379 @default.
• W2790507197 hasConcept C203092338 @default.
• W2790507197 hasConcept C2776356578 @default.
• W2790507197 hasConcept C2776728740 @default.
• W2790507197 hasConcept C2778045022 @default.
• W2790507197 hasConcept C2778558090 @default.
• W2790507197 hasConcept C2779134260 @default.
• W2790507197 hasConcept C2780596555 @default.
• W2790507197 hasConcept C33923547 @default.
• W2790507197 hasConcept C535046627 @default.
• W2790507197 hasConcept C61943457 @default.
• W2790507197 hasConcept C71924100 @default.
• W2790507197 hasConceptScore W2790507197C114614502 @default.
• W2790507197 hasConceptScore W2790507197C126322002 @default.
• W2790507197 hasConceptScore W2790507197C161921814 @default.
• W2790507197 hasConceptScore W2790507197C187212893 @default.
• W2790507197 hasConceptScore W2790507197C197934379 @default.
• W2790507197 hasConceptScore W2790507197C203092338 @default.
• W2790507197 hasConceptScore W2790507197C2776356578 @default.
• W2790507197 hasConceptScore W2790507197C2776728740 @default.
• W2790507197 hasConceptScore W2790507197C2778045022 @default.
• W2790507197 hasConceptScore W2790507197C2778558090 @default.
• W2790507197 hasConceptScore W2790507197C2779134260 @default.
• W2790507197 hasConceptScore W2790507197C2780596555 @default.
• W2790507197 hasConceptScore W2790507197C33923547 @default.
• W2790507197 hasConceptScore W2790507197C535046627 @default.
• W2790507197 hasConceptScore W2790507197C61943457 @default.
• W2790507197 hasConceptScore W2790507197C71924100 @default.
• W2790507197 hasLocation W27905071971 @default.
• W2790507197 hasOpenAccess W2790507197 @default.
• W2790507197 hasPrimaryLocation W27905071971 @default.
• W2790507197 hasRelatedWork W1504654674 @default.
• W2790507197 hasRelatedWork W1573033539 @default.
• W2790507197 hasRelatedWork W2067161789 @default.
• W2790507197 hasRelatedWork W2300951885 @default.
• W2790507197 hasRelatedWork W2560202024 @default.
• W2790507197 hasRelatedWork W2765483614 @default.
• W2790507197 hasRelatedWork W2781904792 @default.
• W2790507197 hasRelatedWork W2789114047 @default.
• W2790507197 hasRelatedWork W2800417626 @default.
• W2790507197 hasRelatedWork W2800528626 @default.
• W2790507197 hasRelatedWork W2801940350 @default.
• W2790507197 hasRelatedWork W2803651213 @default.
• W2790507197 hasRelatedWork W2806319173 @default.
• W2790507197 hasRelatedWork W2811297374 @default.
• W2790507197 hasRelatedWork W2885049414 @default.
• W2790507197 hasRelatedWork W2938881558 @default.
• W2790507197 hasRelatedWork W3018899791 @default.
• W2790507197 hasRelatedWork W3108392184 @default.
• W2790507197 hasRelatedWork W3139620910 @default.
• W2790507197 hasRelatedWork W3208089793 @default.
• W2790507197 hasVolume "88" @default.
• W2790507197 isParatext "false" @default.
• W2790507197 isRetracted "false" @default.
• W2790507197 magId "2790507197" @default.
• W2790507197 workType "article" @default.