Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe neurological disorders, today announced positive interim results from its ongoing POLARIS ...

A new overnight respite facility is opening up in Ipswich which carers have said will be a lifeline. Wots Up, a community ...

Encoded Therapeutics Inc., a clinical-stage genetic medicines company, today announced upcoming presentations of interim clinical data from its ongoing POLARIS Phase 1/2 open-label trials evaluating ...

The heartbroken family of a young man who died while training for a white-collar boxing charity match have spoken out in fury ...

This feature first ran in a special publication called Hometown Pride, published June 28, 2025, featuring people and ...

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, ...

After raising $52,000 with a 5K last year to fund research into their son’s rare seizure disorder, Matthew and Reagan Hill hope for similar success at “Cael’s Climb” at McDade Park on Sunday. Cael ...

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 ...