Gene Therapy

According to results of a recent survey, more than 80% of U.S.-based neurologists agreed that a major unmet need in confronting Huntington’s disease is halting neurodegeneration prior to the onset of symptoms.

Vyjuvek, the first FDA-approved treatment for dystrophic epidermolysis bullosa, has received a permanent J-code from the Centers for Medicare and Medicaid Services, Krystal Biotech announced in a press release.

The Japanese Ministry of Health, Labour and Welfare has granted Vyjuvek an orphan drug designation for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.

This year’s ASH Annual Meeting featured several high-impact abstracts.

Meaningful progress is being made in the development of gene therapies for rare neurological diseases caused by single gene mutations.

A gene therapy company has announced updated interim data from ongoing United States and European phase 1/2 clinical trials of AMT-130 to treat Huntington’s disease.

ORLANDO — While effective medications for epilepsy exist, scientific advancement requires that future gene-targeting therapies or cures benefit the largest number of patients while overcoming several barriers, according to a presentation.

In this video, Lindsey A. George, MD, discussed updated clinical trial data around hemophilia gene therapy presented at ASH Annual Meeting and Exposition.

In this video interview with Healio, Lindsey A. George, MD, spoke about a session on factor VIII biochemistry presented at ASH Annual Meeting and Exposition.

In this video, Lindsey A. George, MD, talked about improving the function of factor VIII variants for hemophilia A gene therapy, which she presented in a session at ASH Annual Meeting and Exposition.