Monday, May 22, 2023

The FDA just approved rub-on gene therapy that helps “butterfly” children

Amazing stuff! Just rub it on! This is almost like a miracle healing!

"Antonio Vento is 13 years old. He’s a tiny figure in bandages who doesn’t walk and, until recently, couldn’t see more than shadows. He has dystrophic epidermolysis bullosa, an inherited disease that makes his skin so fragile that kids with the illness are called “butterfly children.”
But now, thanks to a novel gene therapy squirted onto his skin and dripped into his eyes, things are better. His wounds have gotten smaller, and a visit to the eye doctor this week confirmed that his vision had dramatically improved. ..."

"... How it works: The treatment introduces a missing gene to skin cells so they can make collagen. It’s already helping people with dystrophic epidermolysis bullosa, a rare inherited disease that makes skin incredibly fragile. The topical ointment helps to heal the disease’s chronic, blistering wounds, while an eyedrop version can prevent scar tissue from building up in their eyeballs and improve their vision. ...
Dystrophic Epidermolysis Bullosa (DEB) is a serious rare genetic disease that affects the skin and mucosal tissues and is caused by one or more mutations in the COL7A1 gene, resulting in lack of production of functional type VII collagen (COL7) protein
VYJUVEK is a topical gel that addresses the genetic cause of DEB by restoring functional copies of the COL7A1 gene to patients and is the only medicine available for patients in the US
VYJUVEK is approved for the treatment of patients six months of age or older with either recessive or dominant DEB ..."

Krystal Biotech just got the first approved gene therapy for skin disease | MIT Technology Review Biotech companies are getting creative with how they deliver DNA fixes into people's bodies.


Anthony Vento with his mother and father. His skin disease is being treated with a newly approved gene therapy.


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