Diseases
From The Cure For The Needy
Please click the following links for information on each disease our target molecules treat. Each page has background information on the disease itself, internal links to the current drugs (on our site) that treat the disease, and external links for further reading.
Neglected Tropical Diseases
Leishmaniasis - treated by an orphan drug
Medical Conditions treated by Orphan Drugs
The FDA's list of designated orphan drugs and the diseases they treat:
http://www.fda.gov/orphan/designat/list.htm
- Adrenocortical carcinoma
- Hereditary Angioedema
- Beta-thalassemia (chronic anemias)
- Brain cancer (including glioma, glioblastoma multiforme, medulloblastoma and astrocytoma), multiple
myeloma and lymphoma (Hodgkin's and non-Hodgkin)
- CML: Chronic Myelogenous (or myeloid) leukemia
- Cystinuria
- Epilepsy
- Type 1 Gaucher's disease (GD1)
- Hepatic encephalopathy
- Hyperammonaemia (in patients with N-acetylglutamate synthase deficiency)
- Hyperkinetic movement disorder
- Hyperprolactinemia
- Intestinal infections (such as cryptosporidiosis, amoebiasis and leishmaniasis)
- Ischaemia
- Leishmaniasis
- Mantle cell lymphoma
- Molar pregnancy
- Myelodysplastic syndrome (MDS)
- Pulmonary hypertension
- Raynaud's phenomenon
- Relapsed multiple myeloma
- Scleroderma
- Thrombocytopenia (abnormally low platelet counts)
- Essential thrombocytosis
- Traveler's diarrhea
- Trypanosomiasis
- Hereditary tyrosinemia type 1
- Tuberculosis
- Urea cycle disorders
- Urinary tract infections