ArticleEMBO molecular medicine2017
Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.
Article in EMBO molecular medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis 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
14 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Impact of biobanks on research outcomes in rare diseases: a systematic review.Orphanet journal of rare diseases · 2018Pooled it
- The evolution of lipid nanoparticles: Paving the way for next-generation nucleic acid medicines.Asian journal of pharmaceutical sciences · 2026Review
- Telethon Undiagnosed Disease Program: Structured approach to solving rare childhood-onset genetic diseases.Genetics in medicine open · 2026Article
- Peptide Self-Assembly Facilitating DNA Transfection and the Application in Inhibiting Cancer Cells.Molecules (Basel, Switzerland) · 2024Article
- Unlocking the full potential of rare disease drug development: exploring the not-for-profit sector's contributions to drug development and access.Frontiers in pharmacology · 2024Review
- Article
- Fondazione Telethon and Unione Italiana Lotta alla Distrofia Muscolare, a successful partnership for neuromuscular healthcare research of value for patients.Orphanet journal of rare diseases · 2021Review
- Plant Viruses and Bacteriophage-Based Reagents for Diagnosis and Therapy.Annual review of virology · 2020Review
- An "all-wheel drive" proposal to accelerate clinical research in common and rare neurological diseases.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2020Review
- EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation.Molecular therapy. Methods & clinical development · 2019Review
- Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem.EMBO molecular medicine · 2017Article
- Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.Journal of inherited metabolic disease · 2017Review
- Gene therapy for ADA-SCID, the first marketing approval of anEMBO molecular medicine · 2017Article
- Forkhead-Box-P3 Gene Transfer in Human CD4Frontiers in immunology · 2017Review
Corrections and comments
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Authors and funding
2 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.