Explore rare diseases in clinical trials
Check which rare diseases are currently being frequently studied.
See also our popular diseases glossary and drug database.
Starting by letter
To open the rare diseases glossary, select a letter from the list below.
Rare diseases starting with “u”
- 1
Undifferentiated sarcoma
Undifferentiated sarcoma is a rare type of cancer that develops in the body’s connective tissues, often affecting the arms, legs, or abdomen. This aggressive disease requires prompt diagnosis and a combination of treatments to give patients the best chance of recovery.
- 1
Urea cycle disorder
Urea cycle disorder is a group of rare genetic conditions that prevent the body from removing toxic ammonia from the blood. When ammonia builds up, it can cause serious brain damage and affect organ function, making these disorders life-threatening without proper treatment.
- 2
Usher’s syndrome
Usher’s syndrome is a rare genetic condition that affects both hearing and vision, and is the most common cause of combined deafness and blindness.
- 2
Uterine leiomyosarcoma
Uterine leiomyosarcoma is a rare and aggressive cancer that develops in the muscle wall of the uterus, accounting for only 2% to 5% of all uterine cancers. Despite often being discovered in early stages, this challenging disease requires specialized treatment and long-term monitoring.


