International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Rare Case Studies and Reports

Rare Case Studies and Reports

Case reports, which are defined as scientific documentation of a single clinical observation, have a long and distinguished history in medicine and scientific publication. Case reports are a significant, timely, and relevant study design in the advancement of medical scientific knowledge, particularly in the field of rare diseases. While case study methodology has clear limitations in terms of determining treatment and developing new tests, the observation of a single patient can contribute to our understanding of the aetiology, pathogenesis, natural history, and treatment of particularly rare diseases, as well as the training of future junior investigators. In recent years, this type of scientific publication has come under fire from some in the medical scientific publishing community, and case studies have been pushed to the bottom of the research design hierarchy. Case reports are the simplest form of evidence, but they are also the first line of evidence because they are where new issues and ideas develop. The significance of the observation being reported will be obvious in a strong case report. A medical case report, often known as a case study, is a comprehensive account of a patient encounter. The most crucial part of a case report, and the reason you'd care to write one, is that the situation is sufficiently unique, rare, or intriguing that other medical professionals can learn from it.

Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Vicki Ratner

Vicki Ratner

ESSIC - International Society for the Study of BPS, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Kondakova O. B

Kondakova O. B

National Medical Research Center for Children's Health Federal state autonomous institution of the Russian Federation Ministry of Health, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2023 - Paul L Kaufman

Paul L Kaufman

University of Wisconsin-Madison, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Magali Taiel

Magali Taiel

GenSight Biologics, France

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