Rare Diseases Symptoms Automatic Extraction

Advances in understanding and treating dystrophic epidermolysis bullosa.

[dystrophic epidermolysis bullosa]

Epidermolysis bullosa is a group of inherited disorders that can be both systemic and life-threatening. Standard treatments for the most severe forms of this disorder, typically limited to palliative care, are ineffective in reducing the morbidity and mortality due to complications of the disease. Emerging therapies-such as the use of allogeneic cellular therapy, gene therapy, and protein therapy-have all shown promise, but it is likely that several approaches will need to be combined to realize a cure. For recessive dystrophic epidermolysis bullosa, each particular therapeutic approach has added to our understanding of type VII collagen (C7) function and the basic biology surrounding the disease. The efficacy of these therapies and the mechanisms by which they function also give us insight into developing future strategies for treating this and other extracellular matrix disorders.

Diseases presenting "gene therapy" symptom

  • canavan disease
  • dystrophic epidermolysis bullosa
  • wiskott-aldrich syndrome

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