The number of treatments for rare genetic disorders is expected to increase sharply in the next several years.

NORWELL, Mass., Feb. 23, 2026 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) is calling attention to rare diseases as a significant and growing public health challenge affecting ...

Denny Ladkani lives with Duchenne Muscular Dystrophy, a rare genetic disease that causes progressive muscle degeneration and weakness throughout the body.

Today, the American Kidney Fund (AKF) and the IgA Nephropathy Foundation are on Capitol Hill with more than 80 advocates who are living with rare kidney diseases to urge elected officials to support ...

The National Organization for Rare Disorders (NORD®) is honoring a distinguished group of biopharma industry leaders, medical scientists, and patient advocates for their contributions to improving the ...

On paper, I am rare. In real life, I am the man waiting in OPD queues, googling symptoms at 2 a.m., and nodding politely when someone says, “But you look fine.” Rare feels ordinary — until it doesn’t.

A 25-year-old living with Duchenne muscular dystrophy explains why the Senate confirmation fight over FDA nominee Dr. Heidi ...

Chiesi’s abstract presentations highlight clinical insights and patient-reported outcomes inFabry disease and alpha-mannosidosis ---- Rooted ...

Beacon’s laru-zova gene therapy improved low-light vision in a pivotal XLRP trial, paving the way for discussions with regulators.

Rare diseases present complex coverage challenges under Medicare. Learn how the criteria for FDA approval, genetic testing ...

Canada’s rare disease diagnostic challenge cannot be solved by genomics and data alone; it requires investment in the people, expertise, and clinical systems needed to recognize rare diseases earlier ...

Eric Dube, Ph.D., the CEO who rebranded Travere Therapeutics and oversaw the launch of its rare kidney disease drug Filspari, will ...