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Editorial

Rare diseases represent a major challenge for affected patients, their relatives, physicians, nursing staff, and therapists. For medical and economic reasons, disease rarity complicates the research and medical care of affected patients. The Hollywood movie, “Lorenzo’s Oil”, touchingly illustrates t...

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Autor principal: Plontke, Stefan K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Georg Thieme Verlag KG 2021
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354573/
https://www.ncbi.nlm.nih.gov/pubmed/34352897
http://dx.doi.org/10.1055/s-1397-0832
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author Plontke, Stefan K.
author_facet Plontke, Stefan K.
author_sort Plontke, Stefan K.
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description Rare diseases represent a major challenge for affected patients, their relatives, physicians, nursing staff, and therapists. For medical and economic reasons, disease rarity complicates the research and medical care of affected patients. The Hollywood movie, “Lorenzo’s Oil”, touchingly illustrates the complex problems associated with orphan disease diagnostics, research, and therapy. Directed by George Miller, this film shows the true story of a boy named Lorenzo Michael Murphy Odone, who was diagnosed in 1984 at the age of 6 with the rare neural disease adrenoleukodystrophy (ALD). The movie highlights the manifold problems associated with rare diseases — a large number of which still exist today. However, especially in recent years, orphan diseases have been placed in the focus of public attention.
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spelling pubmed-83545732021-08-11 Editorial Plontke, Stefan K. Laryngorhinootologie Rare diseases represent a major challenge for affected patients, their relatives, physicians, nursing staff, and therapists. For medical and economic reasons, disease rarity complicates the research and medical care of affected patients. The Hollywood movie, “Lorenzo’s Oil”, touchingly illustrates the complex problems associated with orphan disease diagnostics, research, and therapy. Directed by George Miller, this film shows the true story of a boy named Lorenzo Michael Murphy Odone, who was diagnosed in 1984 at the age of 6 with the rare neural disease adrenoleukodystrophy (ALD). The movie highlights the manifold problems associated with rare diseases — a large number of which still exist today. However, especially in recent years, orphan diseases have been placed in the focus of public attention. Georg Thieme Verlag KG 2021-04 2021-04-30 /pmc/articles/PMC8354573/ /pubmed/34352897 http://dx.doi.org/10.1055/s-1397-0832 Text en The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/). https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License, which permits unrestricted reproduction and distribution, for non-commercial purposes only; and use and reproduction, but not distribution, of adapted material for non-commercial purposes only, provided the original work is properly cited.
spellingShingle Plontke, Stefan K.
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title_full Editorial
title_fullStr Editorial
title_full_unstemmed Editorial
title_short Editorial
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url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354573/
https://www.ncbi.nlm.nih.gov/pubmed/34352897
http://dx.doi.org/10.1055/s-1397-0832
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