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

XTalks

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.

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Stoke Therapeutics Announces Presentations Related to the Company’s Work to Advance STK-001, the First Potential New Medicine to Target the Underlying Cause of Dravet Syndrome at the American Epilepsy Society (AES) 2020 Annual Meeting

The Pharma Data

Secondary and exploratory endpoints in the study will evaluate changes in other disease measures, including seizures and additional non-seizure comorbidities. Participants continue to receive their usual care, and will be observed by a team of doctors and nurses over time for up to two years. 4, 2020 14:57 UTC. BEDFORD, Mass.–(