The U.S. Food and Drug Administration approved a gene therapy for dystrophic epidermolysis bullosa, also known as butterfly disease.
A new gene therapy gel is the first-ever approved treatment for the most severe forms of"butterfly disease," a rare and painful skin blistering condition, the U.S. Food and Drug Administration announced Friday .
Due to a genetic mutation, patients cannot make a specific type of collagen in their skin. This collagen would normally anchor different layers of skin to one another and thus prevent them from rubbing against each other and erupting in blisters. The most common side effects included itching, chills, redness, rash, cough and runny nose, according to the FDA.
The gene therapy gel is now approved for use in DEB patients who are at least 6 months old, and the treatment can be applied by a healthcare professional either in a clinic or in the patients' home, according to Krystal Biotech, the sponsor of the trials.
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