- Anämie= Blutarmut
- Chitotriosidase= Spezieller Kontrollwert für den Schweregrad der Gaucher-Krankheit, wird im Blut gemessen
- Gaucher-Zellen = Makrophagen mit gespeichertem Glukozerebrosid
- Glukozerebrosid= Fettstoff, der bei Patienten mit Morbus Gaucher vermehrt gespeichert wird
- Glukozerebrosidase= Enzym, unter dessen Einfluss Glukozerebrosid zu Glukose und Zerebrosid abgebaut wird
- Glukozerebrosid-Synthase= Enzym, unter dessen Einfluss aus Glukose und Zerebrosid Glukozerebrosid gebildet wird
- Hepatosplenomegalie = Vergrößerung von Leber und Milz
- Lysosom= Kleiner Zellbestandteil in Makrophagen, in dem mit Hilfe von Enzymen körpereigene oder fremde Stoffe abgebaut werden
- Makrophage= Sonderform der weißen Blutkörperchen; sog. „Fresszelle“; die Krankheitserreger aufnehmen, verdauen und damit unschädlich machen kann
- Sphingolipid= Fettstoff, der für den Aufbau von Zellwänden wichtig ist
- Thrombozytopenie= Verminderte Zahl der Thrombozyten
- Bioavailability
Describes the extent (usually as a percentage) to which an administered substance reaches the systemic circulation. If a medication is administered intravenously, all of it reaches the blood, so it is 100% bioavailable by definition. If the drug is taken orally, the bioavailability is lower because the drug's absorption is incomplete, and the concentration is reduced before it arrives in the circulatory system.
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- Biochemistry
A scientific discipline involving the study of chemical substances, processes and reactions in living cells, tissues, organs and organisms.
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- Biopharmaceutical company
A biopharmaceutical company uses biological techniques to produce a pharmaceutical product. Actelion is a biopharmaceutical company.
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- Blood-brain barrier
A layer of tightly-packed cells in and around the small blood vessels in the brain that prevent many medications and other substances from entering the brain. It serves to protect the central nervous system.
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- Bone crisis
Also called bone infarction, which means tissue death, in this case in the bones. Tissue dies because of a lack of oxygen as a result of a blocked blood supply.
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- Diastolic pressure
The lowest level to which blood pressure falls between contractions of the ventricles, when the heart relaxes.
- Digital ulcers
Necrotic lesions located on the fingers, a common complication of systemic sclerosis.
Digital ulcers are very painful, and result in difficult-to-heal open sores on fingers and toes. They leave depressed scars and adversely impact the ability to perform work and daily activities, particularly those associated with fingertip functions. In severe cases, infection can become a complication, leading to osteomyelitis and gangrene, for which surgery and even amputation may be required.Related links
- Drug discovery
Research process to identify, select and optimize compounds (biological, genetic or protein targets linked to a particular disease) for clinical investigation.
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- Drug resistance
A description of pathogens when they are unaffected by a drug or when the drug has a reduced effect on them.
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- Dyspnea
Shortness of breath, difficulty breathing.
See also pulmonary arterial hypertension
