Evidence map›Paper›PMID 31704158›Full record

Trial reportNeuromuscular disorders : NMD2019

Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.

Darryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, Wuh-Liang Hwu, Thomas O Crawford, Richard S Finkel, Janbernd Kirschner, Nancy L Kuntz, Julie A Parsons, Monique M Ryan and 20 more

Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neuromuscular disorders : NMD, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03709784 (A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA®), which is not on this map. Cited by 329 papers, 9 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
329citing papers in PubMed, 9 pooled it
44.6field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03709784 active not recruitingnot on this map

A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy

TypeobservationalSponsorWashington University School of MedicineRan2018 to 2025Enrolled148ConditionsSpinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3ArmsObservational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
3 · Its place in the literature

Who cites it

329 citing papers in PubMed, 9 syntheses or guidelines pooled it, 612 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Pooled it
  6. Pooled it
  7. The impact of threeFrontiers in neurology · 2024
    Pooled it
  8. Guideline
  9. Pooled it
  10. Trial
  11. Trial
  12. Review
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Review
  19. A Single-Tube Nested PCR Method forMethods and protocols · 2026
    Article
  20. Review

269 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

30 authors at 18 institutions in 8 countries.

Darryl C De VivoDepartments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, NY 10032, USA. Electronic address: dcd1@cumc.columbia.edu.
Enrico BertiniUnit of Neuromuscular and Neurodegenerative Disorders, Post-Graduate Bambino Gesù Children's Research Hospital, IRCCS, Rome, Italy.
Kathryn J SwobodaDepartment of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.
Wuh-Liang HwuDepartments of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
Thomas O CrawfordDepartment of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Richard S FinkelDivision of Neurology, Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA.
Janbernd KirschnerDepartment of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University of Freiburg, Freiburg, Germany; Department of Neuropediatrics, University Medical Hospital, Bonn, Germany.
Nancy L KuntzAnn & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Julie A ParsonsChildren's Hospital of Colorado, University of Colorado School of Medicine, Aurora, CO, USA.
Monique M RyanRoyal Children's Hospital, University of Melbourne, Murdoch Children's Research Institute, Melbourne, Australia.
Russell J ButterfieldDepartment of Pediatrics and Neurology, University of Utah, Salt Lake City, UT, USA.
Haluk TopalogluDepartment of Pediatric Neurology, Hacettepe University, Ankara, Turkey.
Tawfeg Ben-OmranSidra Medicine, Department of Pediatrics, Qatar Foundation, Doha, Qatar; Division of Clinical and Metabolic Genetics, Department of Pediatrics, Hamad Medical Corporation, Doha, Qatar.
Valeria A SansoneNEMO Clinical Center - NEuroMuscular Omniservice, Milan, Italy; Department of Biomedical Sciences for Health, Università degli Studi di Milano, Milan, Italy.
Yuh-Jyh JongGraduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University; Departments of Pediatrics and Laboratory Medicine, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
Francy ShuDepartment of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
John F StaropoliBiogen, Cambridge, MA, USA.
Douglas KerrBiogen, Cambridge, MA, USA.
Alfred W SandrockBiogen, Cambridge, MA, USA.
Christopher StebbinsBiogen, Cambridge, MA, USA.
Marco PetrilloBiogen, Cambridge, MA, USA.
Gabriel BraleyBiogen, Cambridge, MA, USA.
Kristina JohnsonBiogen, Cambridge, MA, USA.
Richard FosterBiogen, Maidenhead, Berkshire, UK.
Sarah GheuensBiogen, Cambridge, MA, USA.
Ishir BhanBiogen, Cambridge, MA, USA.
Sandra P ReynaBiogen, Cambridge, MA, USA.
Stephanie FradetteBiogen, Cambridge, MA, USA.
Wildon FarwellBiogen, Cambridge, MA, USA.
NURTURE Study Group
Biogen (United States) · USBambino Gesù Children's Hospital · ITBiogen (United Kingdom) · GBCentro Clinico Nemo · ITChildren's Hospital Colorado · USColumbia University Irving Medical Center · USHacettepe University · TRHamad Medical Corporation · QAJohns Hopkins Medicine · USKaohsiung Medical University Chung-Ho Memorial Hospital · TWLurie Children's Hospital · USMassachusetts General Hospital · USNational Taiwan University Hospital · TWNemours Children's Clinic · USRoyal Children's Hospital · AUUniversity Hospital Bonn · DEUniversity of California, Los Angeles · USUniversity of Utah · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety outcomes as of March 29, 2019 in 25 children with genetically diagnosed SMA who first received nusinersen in infancy while presymptomatic in the ongoing Phase 2, multisite, open-label, single-arm NURTURE trial. Fifteen children have two SMN2 copies and 10 have three SMN2 copies. At last visit, children were median (range) 34.8 [25.7-45.4] months of age and past the expected age of symptom onset for SMA Types I or II; all were alive and none required tracheostomy or permanent ventilation. Four (16%) participants with two SMN2 copies utilized respiratory support for ≥6 h/day for ≥7 consecutive days that was initiated during acute, reversible illnesses. All 25 participants achieved the ability to sit without support, 23/25 (92%) achieved walking with assistance, and 22/25 (88%) achieved walking independently. Eight infants had adverse events considered possibly related to nusinersen by the study investigators. These results, representing a median 2.9 years of follow up, emphasize the importance of proactive treatment with nusinersen immediately after establishing the genetic diagnosis of SMA in presymptomatic infants and emerging newborn screening efforts.

Indexed as

Child, PreschoolFemaleFollow-Up StudiesHumansInfantInfant, NewbornMaleMotor ActivityMuscular Atrophy, SpinalOligonucleotidesSurvival of Motor Neuron 2 ProteinTime FactorsTreatment OutcomenusinersenOligonucleotidesSMN2 protein, humanSurvival of Motor Neuron 2 ProteinClinical trialNeurofilamentNewborn screeningNusinersenPresymptomaticSpinal muscular atrophy

Identifiers

PMID31704158
PMCPMC7127286
OpenAlexW2972356268

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.