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Dystrophic Epidermolysis Bullosa

All articles tagged with #dystrophic epidermolysis bullosa

health2 years ago

"Revolutionary Gene Therapy Eyedrops Offer Hope for Restoring Vision to Millions"

Gene therapy eyedrops have successfully restored the sight of a 14-year-old boy with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters in the eyes. The treatment, called Vyjuvek, uses an inactivated herpes simplex virus to deliver working copies of the gene responsible for producing collagen 7, which holds together the skin and corneas. After two years of testing and approvals, the boy's vision significantly improved, and similar therapies could potentially help millions of people with other eye diseases. The gene therapy eyedrops have the potential to be adapted for various conditions, offering hope for future patients.

via Medical Xpress|
#dystrophic-epidermolysis-bullosa#eye-diseases#gene-therapy
health2 years ago

"Revolutionary Gene Therapy Eyedrops Offer Hope for Restoring Vision to Millions"

Gene therapy given through eyedrops has successfully restored the sight of a 14-year-old boy with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters in the eyes. The treatment, called Vyjuvek, uses an inactivated herpes simplex virus to deliver working copies of the gene responsible for producing collagen 7, which holds together both skin and corneas. The therapy not only helped the boy, but also opened the door to potential treatments for millions of people with other eye diseases. The gene therapy eyedrops could be adapted to treat various conditions by changing the gene delivered by the virus, offering hope for future patients.

via The Associated Press|
#clinical-trial#dystrophic-epidermolysis-bullosa#eye-diseases
healthcare2 years ago

FDA approves groundbreaking topical gene therapy for rare skin disease.

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.

via Endpoints News|
#dystrophic-epidermolysis-bullosa#fda#gene-therapy
healthcare2 years ago

FDA approves groundbreaking rub-on gene therapy for rare skin disorder.

The US FDA has approved Krystal Biotech's Vyjuvek, a topical gene therapy for patients with dystrophic epidermolysis bullosa, a rare genetic skin disorder. The therapy is expected to be available in the US in Q3 2023 and is Krystal's first to be approved in the country. The FDA approval is supported by data from early-to-mid-stage and late-stage studies, which showed Vyjuvek completely healed wounds in about 65% of the participants.

via Reuters|
#dystrophic-epidermolysis-bullosa#fda#gene-therapy
health2 years ago

FDA Approves Topical Gene Therapy for Treatment of Skin Condition Causing Persistent Wounds

The FDA has approved Vyjuvek, a topical gene therapy for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. DEB is a rare genetic skin disorder that causes painful and debilitating blisters and wounds. Vyjuvek is a genetically modified herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds. The safety and effectiveness of Vyjuvek was established in a randomized, double-blinded, placebo-controlled study involving a total of 31 subjects with DEB.

via FDA.gov|
#col7a1#dystrophic-epidermolysis-bullosa#fda