Explain what rare diseases are, the concept of their classification, differences in their prevalence, their origin, and issues around the development of therapies for rare diseases.

RARE DISEASES

Define and use in context, or recognise, definitions and applications of each of the items in bold in the text and defined in the Glossary.

Explain what rare diseases are, the concept of their classification, differences in their prevalence, their origin, and issues around the development of therapies for rare diseases.

Perform a database search for aspects of rare diseases.

Use a digital fluorescence microscope to investigate cell structures and functions.

Describe techniques by which genes can be introduced into cells, or a cell’s genome can be modified.

Explain how health science sector incentives and technological advances have increased the development of drugs for rare diseases.

Outline strategies being used to improve the ability to perform clinical trials for rare diseases.

Give examples of cells that are frequently used in research.

Explain why studying rare diseases helps to elucidate normal cellular functions.

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