Mitochondrial disease is a rare and complex genetic disorder that affects people in different ways. There is currently no cure. The Lily Foundation was founded in 2007 by Liz Curtis in memory of her ...

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: ...

OGM Can Resolve Previously Unresolved Rare Disease CasesOGM Can Serve as a Gold Standard Technique for Characterization of SVs OGM Can Complement Sequencing for Better SV Sensitivity and Genomic ...

A 400km cycling challenge between Murcia and Granada aims to raise funds and awareness for an ultra-rare neurodegenerative ...

Rare disease research operates under conditions of extreme complexity. These pressures are driving new approaches to evidence ...

Powassan is a rare, but serious disease. Its rarity presents a challenge to those who study it. Research in a lab setting requires strict safety controls and investments that are hard to come by for ...

Rare diseases affect millions worldwide, yet many patients spend years without answers due to complex symptoms, coding gaps, and limited access to expert care. Advances in genomics, AI, and ...

The Medicines and Healthcare products Regulatory Agency (MHRA) has announced plans to overhaul its approach to therapies for rare diseases. In a position paper, the agency highlighted that rare ...

New medical developments make it possible to treat an increasing number of severe and rare diseases with novel, high-cost pharmaceuticals. At the same time, there are limited resources. The aim of ...

Rare diseases are more than medical curiosities — they’re powerful teaching tools that challenge students to think critically and diagnose creatively. Integrating them into medical education has been ...

Rare diseases can often be difficult to diagnose because their symptoms can overlap with a variety of conditions. One such disease is acute hepatic porphyria (AHP), a rare genetic disease with ...