ArticleEMBO molecular medicine2019
International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases.
Article in EMBO molecular medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 4 of them syntheses that pooled 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.
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
21 citing papers in PubMed, 4 syntheses or guidelines pooled it.
- Zebrafish models for congenital disorders of glycosylation (CDG): a systematic review.Orphanet journal of rare diseases · 2025Pooled it
- The Landscape of Products for Diabetic Peripheral Neuropathy: A Scientific and Patent Systematic Review.Recent patents on biotechnology · 2025Pooled it
- A systematic review of studies that estimated the burden of chronic non-communicable rare diseases using disability-adjusted life years.Orphanet journal of rare diseases · 2024Pooled it
- Genetically guided precision medicine clinical decision support tools: a systematic review.Journal of the American Medical Informatics Association : JAMIA · 2024Pooled it
- Management of patients with rare diseases in the Middle East: challenges & opportunities - insights from the Rare Advocacy Council.Orphanet journal of rare diseases · 2026Article
- Medical device development and innovation for rare and pediatric populations: a global landscape overview.Orphanet journal of rare diseases · 2026Review
- Navigating gastrointestinal challenges in genetic myopathies: Diagnostic insights and future directions.World journal of methodology · 2025Article
- AI-driven virtual cell models in preclinical research: technical pathways, validation mechanisms, and clinical translation potential.NPJ digital medicine · 2025Review
- The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.Nature reviews. Drug discovery · 2025Review
- EURO-NMD registry: federated FAIR infrastructure, innovative technologies and concepts of a patient-centred registry for rare neuromuscular disorders.Orphanet journal of rare diseases · 2024Review
- Antisense oligonucleotides and their applications in rare neurological diseases.Frontiers in neuroscience · 2024Review
- Drug repurposing for rare: progress and opportunities for the rare disease community.Frontiers in medicine · 2024Article
- A disease-specific iPS cell resource for studying rare and intractable diseases.Inflammation and regeneration · 2023Article
- The Precompetitive Space for Drug or Vaccine Development: What Does It Look Like Now and What Could It Look Like in the Future?The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2023Article
- Addressing the need for patient-friendly medical communications: adaptation of the 2019 recommendations for the management of MPS VI and MPS IVA.Orphanet journal of rare diseases · 2022Review
- Reinforcing Collaboration and Harmonization to Unlock the Potentials of Advanced Therapy Medical Products: Future Efforts Are Awaited From Manufacturers and Decision-Makers.Frontiers in public health · 2021Review
- An Objective Approach to Identify Priority Rare Diseases for the Development of Solutions Reducing the Diagnostic Delay Based on French Data.Frontiers in pharmacology · 2021Review
- Barriers and Considerations for Diagnosing Rare Diseases in Indigenous Populations.Frontiers in pediatrics · 2020Review
- Prot2HG: a database of protein domains mapped to the human genome.Database : the journal of biological databases and curation · 2020Article
- The use of genetically humanized animal models for personalized medicine approaches.Disease models & mechanisms · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
Indexed as
Identifiers
What OpenQuestion holds
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.