Cystic fibrosis clinical symptoms

A number of clinical symptoms characterize cystic fibrosis. Predominant among these is the presence of excess sodium chloride in cystic fibrosis patient sweat. Indeed, measurement of chloride levels in sweat remains the major diagnostic indicator of this disease. Another characteristic is the production of an extremely viscous, custard-like mucus in various body glands/organs that severely compromises their function. Particularly affected are ... 

While certain behavioral and nonbehavioral diseases are believed to be monogenic, diseases such as Huntington s, cystic fibrosis, Marfan, and Hirschsprung result in the specified disease, and the outward appearance or result (phenotype) of the disease varies between individuals. For instance, for Marfan syndrome, there is a level below which the mutant protein does not exhibit itself in an outward manner. Most of these diseases have modifier genes that cause modifications in the outward demonstration of the disease and play a key role in the clinical symptoms. Further, the particular metabolic pathways are often varied, with several of the steps being important and the importance of each mechanistic pathway may differ with every individual. 

The rationale for the need of recovery animals was frequently discussed. For drag substances which require chronic (live time) treatment (e.g. oral anti-diabetics, drags for treatment of hypertension, anti-Parkinson drugs, etc.) the question of recovery is less important than in the case of anti-infectives with, in most cases, short treatment periods where mild symptoms of intolerance, e.g. diarrhoea, are observed. However, inclusion of recovery animals is recommended in general because at the stage of development where first clinical studies are conducted, the whole set of indications is not finally known and line extensions can happen. One example is the use of quinolones and other anti-infectives for the treatment of cystic fibrosis. Another example is drugs used for chemotherapy of malignant diseases where recovery has to be studies anyway. 

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