MIDDLETON, Mass., Nov. 6, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Ultragenyx Pharmaceutical ( (RARE)) just ...

According to a recent disclosure with the SEC, Assenagon Asset Management S.A. lifted its holdings in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) by a whopping 439.0% during the second quarter.

Angelman syndrome (AS) is a rare neurodevelopmental disorder caused by the loss of function of the maternally inherited ubiquitin E3 ligase UBE3A gene. AS is characterized by severe symptoms, ...

The initial list of 150 famous people with ties to Florida has been narrowed down to 10 finalists. Readers can vote in the fourth round to select the top five from the remaining candidates. The USA ...

Florida has a long history of attracting the rich, talented and famous, while producing some legends and icons itself. But which one is the most famous? The USA TODAY Network-Florida is continuing its ...

On Tuesday, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Ionis Pharmaceuticals Inc.’s (NASDAQ:IONS) ION582 for Angelman syndrome (AS), a rare neurological ...

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 ...

August saw the death of many notable individuals from across the cultural spectrum. These included actress Loni Anderson, astronaut James Lovell, judge James Caprio and many more. Here’s a partial ...

Broken Hill man Mitch McKenny plans to run from the outback city to Newcastle in 29 days. He aims to raise awareness and funds for Angelman syndrome, a rare genetic disorder. Tradie 'fed up' as ...

Genetic factors that could contribute to people’s risk of developing chronic fatigue syndrome (CFS), also known as myalgic encephalomyelitis (ME), have been identified in a large study. Scientists ...

What is sleeping beauty syndrome? Kleine-Levin syndrome (KLS), also known as “Sleeping beauty syndrome” or “familial hibernation syndrome,” is a neurological disorder that causes sufferers to sleep ...