Ruthie Davi, vice president of statistics and regulatory innovation at Medidata, highlights the value of technology in rare ...

FCS is a hereditary condition that severely impairs the body's ability to break down fats, leading to dangerously elevated triglyceride levels and placing those affected at risk for acute, potentially ...

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.

Rare Disease Day began in 2008, launched in Europe by EURORDIS to shine a light on millions living with conditions that were often misunderstood, misdiagnosed or ignored. Nearly two decades later, the ...

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

Registration has opened for the online "Run for Kids with Rare Diseases" charity race, allowing participants to raise funds ...

BOSTON and NORWELL, Mass., Feb. 23, 2026 /PRNewswire/ -- In recognition of Rare Disease Day, the National Organization for Rare Disorders (NORD®) today announced the launch of the Earn Your Stripes: ...

Playing with his older brothers, 3-year-old Luke Martine appears to be just like any other child. But his parents say as soon as he was born, something seemed wrong. "He started turning blue. Next ...

Under a scientific, ethical and transparent approach, stem cell banking is a considerate move towards sustainability.

IT’S 1123. MORE THAN 300 MILLION PEOPLE WORLDWIDE ARE LIVING WITH A RARE DISEASE, AND MOST STILL DON’T HAVE EFFECTIVE TREATMENTS. BUT BREAKTHROUGHS IN CELL AND GENE THERAPY ARE SLOWLY CHANGING THAT.

From February 24-26, 2026, the EveryLife Foundation for Rare Diseases (EveryLife Foundation) convened hundreds of patients, caregivers, advocates, researchers, and policymakers from 49 states, the ...

Gabby DiSalvo, Cooking on Wheels, preparing Butterfly Pea Tea for the Rare Disease Awareness Dinner at Connecticut Dining Hall. DiSalvo hosts “Cooking on Wheels,” a cooking show on UCTV. Photo ...