Disease · MONDO_0006543
epidermolysis bullosa dystrophica
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. epidermolysis bullosa dystrophica. Diseases, record MONDO_0006543. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0006543
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
- DEB, Dystrophic epidermolysis bullosa, dermolytic epidermolysis bullosa, dystrophic EB
- Open Targets
Drugs9
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| BEREMAGENE GEPERPAVEC | CHEMBL4594328 | Gene | Approval | EMA Human Drugs and FDA | 7 | 3 | 3 | Drugs and targets |
| DABOCEMAGENE AUTOFICEL | CHEMBL4650267 | Gene | Phase 3 | ClinicalTrials.gov | 3 | 1 | 1 | Drugs and targets |
| PRADEMAGENE ZAMIKERACEL | CHEMBL4594312 | Gene | Phase 3 | ClinicalTrials.gov | 3 | 1 | 1 | Drugs and targets |
| FILGRASTIM | CHEMBL1201567 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| LENOGRASTIM | CHEMBL2109128 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| PEGFILGRASTIM | CHEMBL1201568 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| SINECATECHINS | CHEMBL1201723 | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets | |
| TIMBETASIN ACETATE | CHEMBL4594314 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DIACEREIN | CHEMBL41286 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets2
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| CSF3R | Granulocyte colony-stimulating factor receptor | 3 | Granulocyte colony stimulating factor receptor agonist | Phase 2 | https://conductscience.org/library/genes/1441 | Drugs and targets |
| COL7A1 | Collagen alpha-1(VII) chain | 1 | Collagen alpha-1(VII) chain exogenous gene | Approval | https://conductscience.org/library/genes/1294 | Drugs and targets |
Trials7
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 2 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT04491604 | Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB | Phase 3 | 31 | 2023-02-17 | Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa | BEREMAGENE GEPERPAVEC | Its record names the disease | ClinicalTrials.gov |
| NCT03536143 | A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients | Phase 1/2 | 12 | 2023-01-31 | Dystrophic Epidermolysis Bullosa | BEREMAGENE GEPERPAVEC | Its record names the disease | ClinicalTrials.gov |
| NCT03472287 | To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB) | Phase 1 | 11 | 2020-02-10 | Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa | DIACEREIN | Its record names the disease | ClinicalTrials.gov |
| NCT04917874 | A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa | Phase 3 | 47 | Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa | BEREMAGENE GEPERPAVEC | Its record names the disease | ClinicalTrials.gov | |
| NCT06892639 | Evaluation of D-Fi for the Treatment of Wounds Due to DEB | Phase 3 | 32 | Dystrophic Epidermolysis Bullosa | DABOCEMAGENE AUTOFICEL | Its record names the disease | ClinicalTrials.gov | |
| NCT05725018 | A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients | Phase 3 | 12 | Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB | PRADEMAGENE ZAMIKERACEL | Its record names the disease | ClinicalTrials.gov | |
| NCT03578029 | Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa | Phase 2 | 4 | Junctional Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa | TIMBETASIN ACETATE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials2
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Cohort 1 (Adolescents, Adults) vs Cohort 2 (Children): deaths from any cause | 0 of 8 | 0 of 3 | 11 | NCT03472287 | Trial comparisons |
| Cohort 1 (Adolescents, Adults) vs Cohort 2 (Children): serious adverse events | 0 of 8 | 0 of 3 | 11 | NCT03472287 | Trial comparisons |
Papers from its trials3
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0006543
GET https://conductscience.org/api/diseases/v1/MONDO_0006543/drugs