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.

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