Last year, Zilganersen was granted fast track designation by the FDA ... our wholly-owned investigational medicine for Angelman syndrome, which we believe has transformational potential for ...

On February 7, Carlo Rizzuto, Versant Ventures managing director, appeared on CNBC’s ‘Fast Money’ to discuss ... including ION582 for Angelman syndrome, which is on track to start Phase ...

On February 7, Carlo Rizzuto, Versant Ventures managing director, appeared on CNBC’s ‘Fast Money’ to discuss ... including ION582 for Angelman syndrome, which is on track to start Phase ...

NNZ-2591 shows potential in treating neurodevelopmental condition with no approved therapies. Neuren Pharmaceuticals (ASX: NEU) has announced that the US Food and Drug Administration (FDA) has granted ...

The news: Neuren Pharmaceuticals was among the best-performing ASX 200 stocks in morning trade after securing a rare pediatric disease designation from the US Food and Drug Administration (FDA) for a ...

Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide ...

MONTROSE, Mich. (WJRT) - A Mid-Michigan family is raising money to help their 6-year-old boy with a rare genetic disorder. After spending a few months looking for action figures and fun items, Kristin ...

Laughing is one of the better-known traits of Angelman syndrome, a rare neurogenetic disorder that disrupts typical messaging in the brain. A single missing gene leads to the absence of a vital ...