ReviewIntractable & rare diseases research2017
Novel and emerging therapies in the treatment of recessive dystrophic epidermolysis bullosa.
Review in Intractable & rare diseases research, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 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
50 citing papers in PubMed, 1 synthesis or guideline pooled it, 110 citations in OpenAlex.
- A systematic literature review of the disease burden in patients with recessive dystrophic epidermolysis bullosa.Orphanet journal of rare diseases · 2021Pooled it
- FDA approval of prademagene zamikeracel (Zevaskyn): advancing gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) patients.Annals of medicine and surgery (2012) · 2026Article
- Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa.Molecular therapy. Nucleic acids · 2025Article
- Inflammatory modulation by cord blood stem cells prevented digit deformation in recessive dystrophic epidermolysis bullosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Phenotypic Characteristics of a Patient Cohort With Recessive Dystrophic Epidermolysis Bullosa and the Pathogenic Variant c.7485+5G>A in Intron 98 of COL7A1.American journal of medical genetics. Part A · 2025Article
- Clinical and Allelic Heterogeneity in a Small Cohort of Patients with Inherited Epidermolysis Bullosa.International journal of molecular sciences · 2025Article
- A pathogenic COL7A1 variant highlights semi-dominant inheritance in dystrophic epidermolysis bullosa.BMC medical genomics · 2025Article
- Gene hydrogel platforms for targeted skin therapy: bridging hereditary disorders, chronic wounds, and immune related skin diseases.Frontiers in drug delivery · 2025Review
- Novel readthrough agent suppresses nonsense mutations and restores functional type VII collagen and laminin 332 in epidermolysis bullosa.Molecular therapy. Nucleic acids · 2024Article
- Gene-edited cells: novel allogeneic gene/cell therapy for epidermolysis bullosa.Journal of applied genetics · 2024Review
- Identification of novel small molecule-based strategies of COL7A1 upregulation and readthrough activity for the treatment of recessive dystrophic epidermolysis bullosa.Scientific reports · 2024Article
- Unveiling the value of C-reactive protein as a severity biomarker and the IL4/IL13 pathway as a therapeutic target in recessive dystrophic epidermolysis bullosa: A multiparametric cross-sectional study.Experimental dermatology · 2024Article
- Skin Development and Disease: A Molecular Perspective.Current issues in molecular biology · 2024Review
- A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa.Nature communications · 2024Article
- Antiviral drugs prolong survival in murine recessive dystrophic epidermolysis bullosa.EMBO molecular medicine · 2024Article
- Skin Allograft after Bone Marrow Transplantation of Patient with Recessive Dystrophic Epidermolysis Bullosa.Plastic and reconstructive surgery. Global open · 2023Article
- Biomaterial-based gene therapy.MedComm · 2023Review
- Simple and affordable soft brace application in dystrophic epidermolysis bullosa patients.Frontiers in surgery · 2023Article
- ABE8e adenine base editor precisely and efficiently corrects a recurrent COL7A1 nonsense mutation.Scientific reports · 2022Article
- Clinical characteristics, healthcare use, and annual costs among patients with dystrophic epidermolysis bullosa.Orphanet journal of rare diseases · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Epidermolysis bullosa (EB) is a clinically and genetically heterogeneous group of inherited blistering diseases that affects ∼ 500,000 people worldwide. Clinically, individuals with EB have fragile skin and are susceptible to blistering following minimal trauma, with mucous membrane and other organ involvement in some subtypes. Within the spectrum of EB, ∼ 5% of affected individuals have the clinically more severe recessive dystrophic (RDEB) variant with a prevalence of 8 per one million of the population. RDEB is caused by loss-of-function mutations in the type VII collagen gene,
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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.