In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is generally not inherited but instead ...

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that it will present new preclinical ...

Ionis has announced the design for a Phase III trial of ION582, a treatment for Angelman syndrome (AS), following discussions with the FDA.

James Cronin is taking part in the world’s most famous marathon next April and is pledging to raise £10,000 for a charity, ...

Off a rural road in Medina County is a little church in Valley City where a women’s ministry is having a big impact far beyond the four walls of their church.

Ionis Pharmaceuticals (IONS) announced the pivotal Phase 3 study design following successful alignment with the U.S. FDA on ION582, an ...

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

Colin Farrell is doing his bit to help families with adult children with disabilities like his own son to get the support ...

Colin Farrell is doing his bit to help families with adult children with disabilities like his own son to get the support they need.

Baron Funds, an investment management company, released its “Baron Health Care Fund” third quarter 2024 investor letter. A ...

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...