Genetic Disease, Life Science and Nurses - Clinical Research Informer

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

XTalks

MAY 26, 2023

The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in a statement from the company.

Gene Therapy

Gene Therapy Gene Dermatology FDA Approval

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

XTalks

MAY 12, 2023

What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. So, although it’s not a genetic treatment for Rett syndrome, it does act on the core pathophysiology behind Rett syndrome, which is that synaptic loss.

FDA Approval

FDA Approval Genetics Trials Drugs

Clinical Research Informer

Vyjuvek Gets FDA Nod as First Topical Gene Therapy for Rare Skin Disease

After Daybue’s Approval: Acadia Pharmaceuticals’ Next Steps for the Rett Syndrome Community

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