XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

MARCH 3, 2021

Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” said Hylton V. Joffe, MD, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, in a press announcement from the FDA. “The

FDA Approval 64

FDA Approval Genotype Clinical Trials Clinical Trials 64

XTalks

DECEMBER 12, 2023

Additionally, the potential for accelerated approval of therapies via biomarker surrogate endpoints is introducing new complexities into the mix. “In This approval was based on the observed reduction of plasma neurofilament light chain (NfL) in patients who received tofersen.

Trials 111

Trials Clinical Trials Clinical Trials Genetics 111