XTalks

MAY 26, 2023

Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product. DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis.

Gene Therapy 97

Gene Therapy Gene FDA Approval Dermatology 97

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene. Rett syndrome is a neurodevelopmental disorder — not a neurodegenerative disease.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52