Carmen Akras seemed perfectly healthy when she was born in August 2022. She progressed into a loving, beautiful and vivacious ...

Oppenheimer 36th Annual Healthcare Life Sciences Conference February 26, 2026 12:00 PM ESTCompany ParticipantsBrett ...

At the Contemporary Forum, Late-breaking Science and the Rapid Fire Competition, Shrijay Vijayan, Ph.D., MBA, Polaryx Therapeutics’ Chief Scientific and Business Development Officer, presented ...

Good morning, and welcome to Ionis' Fourth Quarter and Full Year 2025 Financial Results Conference Call. As a reminder, this call is being recorded. At this time, I would like to turn the call over to ...

Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), announces it will be ...

It is hoped screening babies’ DNA could detect hundreds more rare, treatable diseases in their first years of life ...

MP Adrian Ramsay vows to raise newborn screening campaign in Parliament after meeting grieving Norfolk mother.

A Ghanaian neuropsychologist is working to combat cultural taboos surrounding disabilities by making education accessible for all ...

Else Kröner-Fresenius-Stiftung awards €400,000 each to clinician-researchers to help them resume their clinical and research work ...

When Swedish toddler Alma Macall was not granted gene therapy in Sweden, her parents travelled to China in 2024 in a final ...

The Cure took home their first Grammys on Sunday, but weren’t able to accept the award in person. Instead, the iconic rockers, including Simon Gallup, Roger O’Donnell, Jason Cooper, Reeves Gabrels, ...

Krabbe disease, also called globoid-cell leukodystrophy, is caused by a deficiency of the lysosomal enzyme β-galactosylceramidase (GALC), resulting from pathogenic variants in both copies of the GALC ...