Ultragenyx has found early success with its experimental treatment for the rare disease Angelman syndrome, supporting the company’s decision to acquire its development partner two years ago.

See more of our trusted coverage when you search. Prefer Newsweek on Google to see more of our trusted coverage when you search. Mothers are often encouraged to trust their instincts, especially ...

A new review sheds light on the complex molecular mechanisms behind Angelman syndrome (AS), a rare neurogenetic disorder, and explores how cutting-edge gene-targeting therapies may offer more ...

Colin Farrell says his personal life has inspired him to launch a new foundation aimed at helping people with intellectual disabilities. The actor, 48, recently launched the Colin Farrell Foundation, ...

CARLSBAD, Calif., July 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the completed multiple ascending dose (MAD) portion of the Phase 1/2 ...

The Nature Index 2026 Research Leaders reveal the leading institutions and countries/territories in the natural sciences, health sciences, applied sciences and social sciences, according to their ...

In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as ...