Rare Diseases Symptoms Automatic Extraction

Systemic protein therapy for recessive dystrophic epidermolysis bullosa: how far are we from clinical translation?

[dystrophic epidermolysis bullosa]

In this issue, Woodley et al. report restoration of anchoring fibril formation and dermal-epidermal adherence in a murine model of recessive dystrophic epidermolysis bullosa (RDEB) by intravenous injection of recombinant human type VII collagen. This work follows a previous report by the same group of the surprising capability of intradermally injected type VII collagen protein to reverse RDEB, and it opens new therapeutic avenues.

Diseases presenting "new therapeutic avenues" symptom

  • dystrophic epidermolysis bullosa

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