Rare Diseases
Rare diseases are illnesses that affect less than one-tenth of one
percent of the population, but are life-threatening or highly disabling.
Rare illnesses may cause disability and reduced quality of life; they may
place a heavy economic burden on patients and their families; and may even
lead to death at a very young age. But because rare diseases affect such
a small number of people, there may be few medical experts available to
provide the care that patients need.
The purpose of this website is to provide patients and family members with
information on three rare diseases:
- Dermatofibrosarcoma protuberans (DFSP)
- Hypereosinophilic syndrome (HES)/ Chronic Eosinophilic Leukaemia (CEL)
- Myelodysplastic/myeloproliferative disorders
These diseases result from alterations to a part of the body's
blood plasma called growth factors, or to enzymes in the body called kinases.
When these growth factors or kinases are altered, or mutated, they send
out signals, or instructions, for cells in the body to grow abnormally,
causing tumors or other problems.
Click on any of the options on this page for useful information
about the disorder, current treatment options, and helpful resources.
You will also find information about clinical studies of new treatments
for the disorder and ways to decide if being part of one of the studies
is right for you.
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