American Kidney Fund and IgA Nephropathy Foundation Advocate on Capitol Hill for Rare Kidney Disease Legislation

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 ...

Boy, 6, wrote 'Chocolate Bar' for friend with rare liver disorder; book raised $1 million

At 6, Los Angeles boy Dylan Siegel wrote and sold a book calledChocolate Barto help fund research into his best friend ...

Boy, 13, Diagnosed with Rare Genetic Disease Has to Consume Raw Cornstarch to Survive (Exclusive)

While Jen McGowan describes her son as a “really happy baby,” she admits to PEOPLE that his younger years were “intense” ...
Comic Basics

Emma Heming Willis Marks World FTD Awareness Week With a Rare Photo of Bruce Willis (71)

Frontotemporal dementia is still far less understood than Alzheimer's disease, which is why awareness weeks matter so much to ...

I'm 25 with a terminal disease. We need an FDA commissioner that hears people like me

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

Top U.S. Health Officials to Address Rare Disease Leaders at 2026 NORD Breakthrough Summit in Washington, D.C.

Three of the nation's leading federal health officials will join more than 900 rare disease leaders across patient advocacy, industry, policy, science and medicine at the 2026 NORD® Rare Diseases and ...

Hendersonville woman raises awareness for rare liver disease beyond PBC awareness month

Primary biliary cholangitis, or PBC, affects less than 1% of the population. Doctors do not know exactly what causes it, and ...
Opinion
Open Access GovernmentOpinion

Building rare disease diagnosis in Canada: People, expertise, and systems

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 ...

American Kidney Fund’s UCKD Summit Helps Drive Early Recognition for Rare and Genetic Kidney Diseases

Today, the American Kidney Fund (AKF) is convening its seventh annual national summit on Unknown Causes of Kidney Disease (UCKD) in Washington, D.C., bringing healthcare professionals, researchers, ...
BIOENGINEER

Virtual Support Sessions measurably Lift Quality of Life in Autoimmune Blistering Disease Patients

For people living with rare autoimmune blistering diseases, the daily reality extends far beyond the blisters themselves.
Public Broadcasting Service (PBS)

How a rare genetic mutation could unlock new ways to treat or prevent dementia

Over 55 million people worldwide are living with the devastating reality of dementia, but researchers have now identified ...

MavriX Bio Announces FDA Rare Pediatric Disease Designation for Investigational MVX-220 for the Treatment of Angelman Syndrome

AS2Bio: MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the U.S. Food and Drug ...