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 “o”

  • 1

    Ocular melanoma

    Ocular melanoma is the most common eye cancer in adults and the second most common type of melanoma after skin melanoma, yet it remains largely unknown to the public despite affecting approximately 2,000 adults each year in the United States.

  • 1

    Optic glioma

    Optic glioma is a slow-growing brain tumor that develops along the optic nerve, the pathway that carries visual signals from the eye to the brain. While this is a serious condition, it has a high cure rate and primarily affects young children, with most cases diagnosed before the age of 5.

  • 1

    Ornithine transcarbamoylase deficiency

    Ornithine transcarbamoylase deficiency is a rare inherited disorder that prevents the body from safely removing ammonia from the blood, potentially causing severe brain damage if ammonia levels become too high.

  • 8

    Osteosarcoma

    Osteosarcoma is a type of bone cancer that most often affects teenagers and young adults, though it can occur at any age. It typically develops in the long bones of the arms and legs, especially near the knee, and can cause pain, swelling, and limited movement.

  • 5

    Osteosarcoma metastatic

    Metastatic osteosarcoma is when bone cancer spreads from its original location to other parts of the body, most commonly the lungs. This advanced form of bone cancer presents significant treatment challenges and has a less favorable outlook than cancer that remains in one place.

  • 1

    Osteosarcoma recurrent

    Recurrent osteosarcoma is cancer that has returned after initial treatment or has not responded to therapy. It occurs in 30-50% of patients who initially had cancer confined to one area, and in 80% of patients who had cancer that had already spread at diagnosis.