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Rare diseases - Maria Beltran
Maria Beltran
Created on March 23, 2024
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Transcript
Maria Beltran Poy
Medicine AND rARE DISEASES
25.04.24
Sources: eurodis.org, Wikipedia, OMS, Rare diseases Org., World
Introduction
Sandra's Story
INDEX
Video
Rare diseases
Conclusion
Clinical Trials
Introduction
What's medicine?
Health science dedicated to prevention
Rare diseases
Conditions affecting a small percentatge of population They affect 10% of the total population
Purpose of the presentation
Understanding of rare diseases
Rare diseases
What are they?
- Wide diversity of symptoms
Huntington's diseaseCystic fibrosisDuchenne Muscular DystrophyALS (Amyotrophic Lateral Sclerosis)Marfan Syndrome
- Due to low prevalence
- Medical expertise =RARE
- Knowledge = SCARCE
- Care offerings = INADEQUATE
- Reaserch = LIMITED
Examples
Rare diseases and their cures
- 8000 rare diseases
- Only 500 treatments, not cures
- More than 1000 clinical trials
Current Clinical Trials
Clinical Trials
What are they?
- Research studies
- Potential treatments or interventions
- Crucial for developing
Huntington's Disease
Cystic Fibrosis
Sandra's Story
Her life with ALS
Sandra's Story
Her life with ALS