March 12 (Reuters) - Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a late-stage trial to better control ammonia levels linked to a rare inherited disorder ...

Detailed price information for Abeona Therapeutics (ABEO-Q) from The Globe and Mail including charting and trades.

Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks ...

World Kidney Day 2026: here’s how genetic testing is improving diagnosis and treatment of steroid-resistant nephrotic syndrome in children and helping protect long term kidney health.

Now, Chinese scientists have developed a kind of "double-sided tape" that helps the body's own soldiers — engineered immune cells — hold onto cancer cells more tightly, making treatment far more ...

Care hurdles now add to the disease burden faced by U.S. sickle cell patients, despite the promise of emerging options like gene therapy.

My journey has been a long one. I am a paediatric immunologist and principal investigator of a PID research laboratory, and ...

After years on SSRIs, one man shares what it’s like to live with genital numbness, delayed orgasm, and a changed sex life.

Late last year, I nearly passed out in my girlfriend’s bed. I had forgotten to breathe for what felt like a couple of minutes until, finally, I gasped, gulping hot air. My balls ached. My temples ...

Jane Lapotaire, the actress, who has died aged 81, rose to fame in the late 1970s; her large, dark eyes, strong features and husky voice made her the ideal choice for a succession of passionate, ...

Dravet syndrome affects about 1 in 17,500 children. Having two daughters with the condition is “extraordinarily uncommon.” ...

PGN-EDODM1, an experimental DM1 therapy from Pepgen, showed biomarker activity and splicing correction in an early clinical ...