ArticleJournal of comparative effectiveness research2020
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.
Open access · hybridAbstract readMulticenter Study
In one paragraph
Article in Journal of comparative effectiveness research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 55 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
55citing papers in PubMed, 2 pooled it
7.3field-weighted citation impact, top 2% of its field
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.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
55 citing papers in PubMed, 2 syntheses or guidelines pooled it, 126 citations in OpenAlex.
- Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.Journal of neuromuscular diseases · 2024Pooled it
- Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis.Muscle & nerve · 2022Pooled it
- Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.Journal of comparative effectiveness research · 2025Trial
- Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.Journal of comparative effectiveness research · 2022Trial
- Genetic, Clinical, and Management Characteristics of Duchenne Muscular Dystrophy in Saudi Arabia.Healthcare (Basel, Switzerland) · 2026Article
- Skipping the Biopsy: Real-World Experience of Whole-Exome Sequencing as First-Tier Testing in Pediatric Muscular Disorders.International journal of molecular sciences · 2026Article
- Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series.Nature communications · 2025Article
- A precision medicine approach to primary immunodeficiency disease: Ataluren strikes nonsense mutations once again.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Development of translational read-through-inducing drugs as novel therapeutic options for patients with Fanconi anemia.Cell death discovery · 2025Article
- A New Perspective on Drugs for Duchenne Muscular Dystrophy: Proposals for Better Respiratory Outcomes and Improved Regulatory Pathways.Paediatric drugs · 2025Review
- Evidence Following Conditional NICE Technology Appraisal Recommendations: A Critical Analysis of Methods, Quality and Risk of Bias.PharmacoEconomics · 2024Article
- Safety and effectiveness of ataluren in patients with Duchenne muscular dystrophy: single-center experience from Saudi Arabia.The Journal of international medical research · 2024Article
- Transition and management of patients with Duchenne Muscular Dystrophy: a narrative review based on Italian experts' opinion and real-world experience.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024Review
- Gene therapy for ultrarare diseases: a geneticist's perspective.Journal of biomedical science · 2024Review
- The BALB/c.mdx62 mouse exhibits a dystrophic muscle pathology and is a model of Duchenne muscular dystrophy.Disease models & mechanisms · 2024Article
- Article
- The complex landscape of DMD mutations: moving towards personalized medicine.Frontiers in genetics · 2024Review
- Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2024Article
- Review
- Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.Journal of neurology · 2023Article
Corrections and comments
- Commented on by
Authors and funding
17 authors at 9 institutions in 6 countries.
Eugenio MercuriDepartment of Pediatric Neurology, Catholic University, Rome, Italy.
Francesco MuntoniDubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London, WC1N 1EH, UK.
Andrés Nascimento OsorioHospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain.
Már TuliniusDepartment of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
Filippo BuccellaParent Project Italy APS, Rome, Italy.
Lauren P MorgenrothTherapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA.
Heather Gordish-DressmanCenter for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA.
Joel JiangPTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
Panayiota TrifillisPTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
Jin ZhuPTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
Allan KristensenPTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
Claudio L SantosPTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA.
Erik K HenricsonUniversity of California Davis School of Medicine, Davis, CA, USA.
Craig M McDonaldUniversity of California Davis School of Medicine, Davis, CA, USA.
Isabelle DesguerreAPHP Necker - Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France.
STRIDE
CINRG Duchenne Natural History Investigators
PTC Therapeutics (United States) · USUniversity of California, Davis · USAssistance Publique – Hôpitaux de Paris · FRChildren's National · USDrottning Silvias barn- och ungdomssjukhus · SEGreat Ormond Street Hospital for Children NHS Foundation Trust · GBHospital Sant Joan de Déu Barcelona · ESParent Project Onlus · ITUniversità Cattolica del Sacro Cuore · IT
Funding
WU INSTITUTE OF CLINICAL AND TRANSLATIONAL SCIENCESUL1TR002345 · NCATS · WASHINGTON UNIVERSITY · PI William G. Powderly · 2017 to 2026
$97.8MWashington University Institute of Clinical and Translational Sciences (UL1)UL1RR024992 · NCRR · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2007 to 2011
$45.4MSUPPLEMENT FOR RENOVATION OF ANIMAL FACILITIES AT INSTITUTE OF NEUROBIOLOGYG12RR003051 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI RODRIGUEZ-MEDINA, JOSE R · 1986 to 2011
$28.1MTRCLU54RR026139 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI FRONTERA, WALTER R. · 2010 to 2011
$8.4MCTSA INFRASTRUCTURE FOR PEDIATRIC RESEARCHUL1RR031988 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI JOSEPH, JILL G · 2010 to 2011
$8.2MWellstone Muscular Dystrophy Center: Children's Nat'l Med CtrU54HD053177 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI HOFFMAN, ERIC P. · 2005 to 2009
$7.3MBiomarker discovery and validation in a Duchenne dystrophy natural history studyR01AR062380 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HATHOUT, YETRIB, MCDONALD, CRAIG M. · 2011 to 2014
$1.5MClinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic TrialsR01AR061875 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI MCDONALD, CRAIG M. · 2011 to 2014
$1.3MNCATS NIH HHS UL1 TR002345NCRR NIH HHS G12 RR003051NCRR NIH HHS U54 RR026139NCRR NIH HHS UL1 RR024992NCRR NIH HHS UL1 RR031988NIAMS NIH HHS R01 AR061875NIAMS NIH HHS R01 AR062380NICHD NIH HHS U54 HD053177
Abstract
PubMed holds no abstract for this paper.
Indexed as
Codon, NonsenseDystrophinHumansMuscular Dystrophy, DuchenneOxadiazolesRegistriesTreatment OutcomeatalurenCodon, NonsenseDMD protein, humanDystrophinOxadiazolesatalurendystrophineffectivenessnonsense mutation Duchenne muscular dystrophysafetySTRIDE Registry
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390
Registered trials
None linked
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.