Pilot corpus: NIH-funded biomedical datasets. Any DOI can be scored on demand — network effects sharpen as coverage grows.

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

Molecular Genetics and Metabolism(2022)10.1016/j.ymgme.2022.07.017Source: DataRank Database

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study is a research paper published in Molecular Genetics and Metabolism (2022). On theSindex it has a DataRank of 0.978. It has been cited 49 times, with 21 citing works in its 1-hop citation network.

N/A

0.978DataRank · unranked

Scored on demand from live citation data

Deposits data Open Access49 citations · 21 citing works in its network

Download PDF

Cite:

Linked data & code

nct

Data availability

Repositories this paper deposited data in (declared in PubMed).

ClinicalTrials.govNCT02055118

DataRank reads this dataset's downstream impact straight off the citation graph — no black box, no proprietary weighting. How is this computed?

›Methodology & internals

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Pipeline:MetadataData-paper checkEnrichmentCitation networkScoring

Enrichment:Funding (7 grants)Impact (FWCI 4.96)OA: hybridTopics (38)Linked Datasets (16)DataCite Reuse (16)IDs (PubMed, PMC, OpenAlex)Annotations (1)SDGs (2)

FAIR Checklist

Context only (not used in score)

Findable (2/2)

Has DOI
Indexed in repositories

Accessible (1/2)

Open Access

Interoperable (2/2)

DataCite relations
Linked datasets

Reusable (0/3)

FAIR checklist signals are shown for context only and do not affect DataRank scoring.

Run a calibrated FAIR evaluation for this paper →

We only score data papers we can read in full — never from an abstract alone.

DataRank Breakdown

Base Score 60%Citation Network 40%

Base Score Contribution

0.587

From this paper's citation signal

Citation Network Contribution

0.391

From 14 citing papers with measurable signal

Learn more about DataRank methodology →

Top 5 citers driving the network score

Ranked by each citer's contribution to N(p) — log1p(C_q) divided by its reference count — out of 21 citers.

Heparan sulfate in cerebrospinal fluid as a biomarker to assess disease severity and for treatment monitoring in patients with Mucopolysaccharidosis Type II: a position statement
Orphanet Journal of Rare Diseases20246 citations18 referencesContributes 0.108
Intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II: Final report of 5-year results from a Japanese open-label phase 1/2 study
Molecular Genetics and Metabolism202320 citations37 referencesContributes 0.082
Intracerebroventricular Enzyme Replacement Therapy in Patients with Neuropathic Form of Mucopolysaccharidosis Type II: to Help Practicing Physician
Вопросы современной педиатрии20241 citations15 referencesContributes 0.046
Gene therapies for mucopolysaccharidoses
Journal of Inherited Metabolic Disease202321 citations86 referencesContributes 0.036
Safety, tolerability, and pharmacokinetics of verenafusp alfa in healthy volunteers: results of an open-label multicohort phase I study
Terapevticheskii arkhiv20261 citations24 referencesContributes 0.029

Authors (14)

Barbara K. Burton,Paul Harmatz ORCID,Luis González Gutiérrez-Solana ORCID,Matilde Ruíz-Garcìa ORCID,Simon Jones ORCID

Live enrichment

PubMed:36027721 PMC:PMC10826424 OpenAlex:https://openalex.org/W4289525295

NCATS NIH HHS

Grant: UL1 TR002489

NCATS NIH HHS

Grant: UL1 TR001878

NCATS NIH HHS

Grant: UL1 TR002548

National Institutes of Health

Grant: 3UL1TR001878-06S1

Institutional Clinical and Translational Science Award

National Institutes of Health

Grant: 3UL1TR002548-03S1

Clinical and Translational Science Collaborative of Cleveland

National Institutes of Health

Grant: 3UL1TR002489-02S1

North Carolina Translational and Clinical Science Institute (NC TraCS)

Canadian Institutes of Health Research

Grant: unidentified

unidentified

FWCI

4.96

Citation Percentile

1.0%

Citation Trend

2022

2023

2024

2025

2026

hybridLicense: cc-by

journalhttps://doi.org/10.1016/j.ymgme.2022.07.017

publisherhttps://doi.org/10.1016/j.ymgme.2022.07.017

publisherhttps://api.elsevier.com/content/article/PII:S1096719222003845?httpAccept=text/xml

publisherhttps://api.elsevier.com/content/article/PII:S1096719222003845?httpAccept=text/plain

repositoryhttps://pubmed.ncbi.nlm.nih.gov/36027721

Fields of Study

Lysosomal Storage Disorders ResearchFamily and Disability Support ResearchGlycogen Storage Diseases and Myoclonus0301 basic medicine03 medical and health sciences0302 clinical medicine

MeSH Terms

ChildChild, PreschoolGlycosaminoglycansHumansIduronate SulfataseIduronic AcidMultiple MyelomaMucopolysaccharidosis IIEnzyme Replacement Therapy

Keywords

Mucopolysaccharidosis type IIHunter syndromeClinical endpointMedicineRandomized controlled trialInternal medicineEnzyme replacement therapyDiseaseCognitive impairmentIntrathecalMps IiMucopolysaccharidosis IiIdursulfaseIduronic Acid610Iduronate SulfataseArticleChild, PreschoolHumansPreschoolChildMultiple MyelomaGlycosaminoglycans

Sustainable Development Goals

SDG 3: 3. Good healthSDG 0: Good health and well-being

Additional file 1 of A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT

10.6084/m9.figshare.24483554.v1figshare

10.6084/m9.figshare.24483554figshare

Additional file 2 of A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT

10.6084/m9.figshare.24484219.v1figshare

10.6084/m9.figshare.24484219figshare

Additional file 2 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.25378199.v1figshare

Additional file 2 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.25378199figshare

Additional file 1 of Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment

10.6084/m9.figshare.25596417.v1figshare

Additional file 1 of Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment

10.6084/m9.figshare.25596417figshare

Additional file 3 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691466.v1figshare

Additional file 3 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691466figshare

Additional file 4 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691469figshare

Additional file 4 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691469.v1figshare

Additional file 5 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691472.v1figshare

Additional file 5 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.26691472figshare

Additional file 1 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.25378010.v1figshare

Additional file 1 of Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

10.6084/m9.figshare.25378010figshare