Fondazione Telethon ETS' Waskyra has become the first gene therapy in the US for patients with Wiskott-Aldrich syndrome (WAS) ...
Lindberg had Parkinson’s disease, the neurologist said, with all the classic symptoms. PD—as the scientists she would meet ...
Sperm from a donor who unknowingly carried a gene that raises the risk of cancer was used to conceive almost 200 children across Europe. A major investigation revealed the sperm was not sold to UK ...
Scientists at UCL and GOSH have used groundbreaking base-edited CAR‑T cell therapy — BE‑CAR7 — to treat aggressive T‑cell ...
Are we ready to live decades longer?
A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...
The Georgia case was brought on behalf of transgender inmates by the Center for Constitutional Rights after Georgia enacted a ...
The U.S. Food and Drug Administration said on Tuesday it had approved the first gene therapy for a rare and life-threatening ...
Clinician Perspectives on Holistic Management of PKP2-Associated Arrhythmogenic Cardiomyopathy December 9, 2025 3:00 ...
Zevaskyn, the first gene therapy for recessive dystrophic epidermolysis bullosa, showed significant wound healing in phase 3 ...
Scientists anticipate a future where transgender people could create their own gender-affirming hormones and even produce ...
Casgevy safely and effectively prevents vaso-occlusive crises in children ages 5 to 11 with severe sickle cell disease, trial ...
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