FDA OKs first-ever topical gene therapy, for rare skin disease
"Vyjuvek is the first FDA-approved gene therapy treatment for DEB, a rare and serious genetic skin disorder," said Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research,"With the FDA approval of Vyjuvek, the DEB population has reached a monumental milestone in the treatment of this horrible disorder," said Brett Kopelan, executive director of debra of America, a national advocacy group for people with...
"Because it's safe and easy to apply directly to wounds, it doesn't require a lot of supporting technology or specialized expertise, making Vyjuvek highly accessible even to patients who live far away from specialized centers," he said. Kopelan, the patient advocate, said that his organization"will continue to work closely with Krystal to assure patients have ready access to Vyjuvek."affects 1 to 3 people per million in the United States. It is caused by mutations in thegene, which encodes the alpha-1 chain of collagen type VII protein. C7 forms the anchoring fibrils that attach the epidermis to the underlying dermal connective tissue.
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