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 ...

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 ...

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 ...

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.

MANILA, Philippines — Growing up, Araceli Lanorio was very thin. As the youngest of six siblings, her family often remarked on her lanky frame but thought nothing of it. It was only when she was in ...

Minimal Symptom Expression (MSE) is defined as a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1, a definition increasingly recognized and applied in MG research and clinical ...

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

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

BRADENTON, Fla. — It’s a disease that’s compared to ALS — slowly weakening the muscles, leaving the patient without the ability to walk, use their hands, and eventually their ability to swallow. It’s ...

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

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

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 ...