Rare pulmonary disease and orphan drugs: a path to the future
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DOI: 10.1183/16000617.0115-2019
Abstract: In this issue of the European Respiratory Review (ERR), we are delighted to publish a series of articles on rare pulmonary diseases discussing hot topics presented at the 8th International Meeting on Pulmonary Rare Diseases… read more here.
Keywords: rare pulmonary; disease orphan; orphan drugs; european respiratory ... See more keywords
Gaucher disease: An orphan disease with significant osseous manifestations
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DOI: 10.15761/jcmm.1000110
Abstract: Gaucher disease, the most common of the lysosomal storage diseases, is a systematic familial disease classified in “orphan diseases’’, a group of rare disorders with prevalence of 1:50,000 or lower in the general population. Gaucher… read more here.
Keywords: gaucher disease; disease orphan; gaucher cells; disease ... See more keywords