Epidermolysis bullosa (EB) is a group of rare inherited skin disorders that cause the skin to become very fragile. Any trauma or friction to the skin can cause painful blisters.1
RDEB and JEB are the two main types of EB.
Infants are typically born with widespread blistering and areas of missing skin, often caused by trauma during birth
Most RDEB patients develop skin cancer by age 35
Average mortality of patients with JEB is 18 months
Affects ~4,000 patients worldwide with nonsense mutations
There are no approved treatments; management is palliative
Preclinical results support that RMAs can restore
functional collagen protein at levels comparable
to high-dose gentamicin
Reference: 1. Genetics home reference: dystrophic epidermolysis bullosa. National Institutes of Health: US National Library of Medicine. https://ghr.nlm.nih.gov/condition/dystrophic-epidermolysis-bullosa#genes. Accessed June 17, 2021.
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