Molecular therapeutics in development for Epidermolysis bullosa: update 2020
Abstract: Epidermolysis bullosa (EB) is a group of rare genetic disorders for which significant progress has been achieved in the development of molecular therapies in the last few decades. Such therapies require knowledge of mutant genes and specific mutations, some of them being allele specific. A relatively large number of clinical trials are ongoing and ascertaining the clinical efficacy of gene, protein or cell therapies or of repurposed drugs, mainly in recessive dystrophic EB. It is expected that some new drugs may emerge in the near future and that combinations of different approaches may result in improved treatment outcomes for individuals with EB
- Location
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Deutsche Nationalbibliothek Frankfurt am Main
- Extent
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Online-Ressource
- Language
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Englisch
- Notes
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Molecular diagnosis & therapy. - 24, 3 (2020) , 299-309, ISSN: 1179-2000
- Event
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Veröffentlichung
- (where)
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Freiburg
- (who)
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Universität
- (when)
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2020
- Creator
- DOI
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10.1007/s40291-020-00466-7
- URN
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urn:nbn:de:bsz:25-freidok-1659682
- Rights
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Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
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14.08.2025, 10:52 AM CEST
Data provider
This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Associated
- Has, Cristina
- South, Andrew
- Uitto, Jouni
- Universität
Time of origin
- 2020
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Homozygous nonsense mutation and additional deletion of an amino acid in BPAG1e causing mild localized Epidermolysis Bullosa simplex
A silent COL17A1 variant alters splicing and causes junctional epidermolysis bullosa
The position of targeted next-generation sequencing in epidermolysis bullosa diagnosis
Successful multidisciplinary treatment of chronic facial wounds in junctional epidermolysis bullosa
Betulin-based oleogel to improve wound healing in dystrophic epidermolysis bullosa: a prospective controlled proof-of-concept study
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Dupilumab improves itch and blistering in different subtypes of epidermolysis bullosa
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Orale Manifestationen bei Epidermolysis bullosa junktionalis: Genotyp-Phänotyp Korrelationen
Homozygous nonsense mutation and additional deletion of an amino acid in BPAG1e causing mild localized Epidermolysis Bullosa simplex
A silent COL17A1 variant alters splicing and causes junctional epidermolysis bullosa
The position of targeted next-generation sequencing in epidermolysis bullosa diagnosis
Successful multidisciplinary treatment of chronic facial wounds in junctional epidermolysis bullosa