The number of clinical trials for rare diseases is increasing drastically from the last ten years to more than 500 as compared to a few hundred clinical trials. Currently, more than 400 orphan designated drugs are commercially available, and around 1000 drugs are undergoing clinical trials. Drug development in rare diseases is often challenging and […]

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For the approximately 7,000 known rare diseases, less than 10% of diseases have an FDA-approved treatment available. Due to a small number of patients, limited understanding of the variability and progression of the diseases, developing a drug or treatment for rare diseases is often challenging. Rare diseases currently affect 3.5% – 5.9% of the worldwide […]

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