Novel and emerging therapies in the treatment of recessive dystrophic epidermolysis bullosa.

No abstract is available for this paper.

• Publication year

  2017

• Venue

  Intractable & Rare Diseases Research

• Publication date

  2017-02-28

• Fields of study

  Medicine

• Identifiers
  DOI 10.5582/irdr.2017.01005PMID 28357176PMCID PMC5359356
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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  2008cited by this paper
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  2008influential reference
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• Bibliometric Analysis of the 50 Most Cited Publications in Epidermolysis Bullosa

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• Phenotypic Characteristics of a Patient Cohort With Recessive Dystrophic Epidermolysis Bullosa and the Pathogenic Variant c.7485+5G>A in Intron 98 of COL7A1

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• A pathogenic COL7A1 variant highlights semi-dominant inheritance in dystrophic epidermolysis bullosa

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• FDA approval of prademagene zamikeracel (Zevaskyn): advancing gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) patients

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• Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa

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• Gene-edited cells: novel allogeneic gene/cell therapy for epidermolysis bullosa

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• Deficient skin proteins rescue of expression in patients with epidermolysis bullosa: efficacy of gentamicin

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