FDA approves groundbreaking topical gene therapy for rare skin disease.
TL;DR Summary
The FDA has approved Krystal Biotech's gene therapy, Vyjuvek, for treating patients with dystrophic epidermolysis bullosa (DEB), a rare genetic disease that causes the skin to be as fragile as a butterfly's wings. The therapy is the first of its kind and offers a long-awaited solution for families afflicted by the condition, for which there are no approved treatments in the US.
Topics:business#dystrophic-epidermolysis-bullosa#fda#gene-therapy#healthcare#krystal-biotech#rare-disease
- FDA approves Krystal Biotech's gene therapy for dystrophic epidermolysis bullosa – Endpoints News Endpoints News
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- The FDA just approved rub-on gene therapy that helps “butterfly” children MIT Technology Review
- Rare Skin Blistering Condition Gets First Drug Approved Medpage Today
- FDA Approves First-Ever Topical Gene Therapy Medscape
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