Challenges in Rare Diseases
There are 7,000 or more uncommon diseases. These rare diseases typically have a hereditary foundation, commonly strike patients as children, are progressive, and frequently pose a serious threat to life. Children who are afflicted with these disorders may have psychological effects from these traits that are quite damaging. Research and development must focus on how individuals with rare diseases' distinctive traits affect clinical development and how those processes interact with regulatory procedures. The fact that the pathophysiology of diseases is still largely unknown presents a basic obstacle in the development of drugs for rare diseases. In addition to these difficulties, the majority of uncommon diseases have no treatments.
- Patient attrition
- Interface between clinical development and the regulatory process
- Less knowledge on pathophysiology of disease
Related Conference of Challenges in Rare Diseases
September 22-23, 2025
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Challenges in Rare Diseases Conference Speakers
Recommended Sessions
- Challenges in Rare Diseases
- Clinical Research on Rare Diseases
- Future of Rare Diseases Research
- Immune System Diseases
- Infectious Disease
- Living with Rare Diseases
- Mystery Diagnosis of Rare Diseases
- Orphan Drugs
- Rare Autoimmune Diseases
- Rare Cancer Diseases
- Rare Dermatological Diseases
- Rare Diseases
- Rare Diseases in Aging
- Rare Diseases in Brain
- Rare Diseases in Endocrine System
- Rare Gastrointestinal Diseases
- Rare Genetic Diseases
- Rare Haematological Diseases
- Rare Pediatric Diseases
- Treatment for Rare Diseases
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