Global trials are critical for developing orphan drugs because they provide the necessary platform to evaluate the safety, efficacy, and overall impact of these therapies on rare disease populations worldwide. Orphan drugs are specifically designed to treat rare diseases and face unique challenges during development, particularly due to the limited number of patients available for […]

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Clinical trials in rare diseases present more significant challenges compared to trials in more common diseases. These challenges arise due to various factors, including trial design, the limited patient population for recruitment, regulatory support, and more. However, implementing a well-defined strategy for clinical development in rare diseases can help overcome these challenges. Such a strategy […]

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Many people with rare diseases struggle to find the right diagnosis and right treatment for years. The lack of awareness about these conditions makes it much harder for them to get the right treatment. Rare Disease Day is all about raising awareness for rare diseases. And, this year, you can join these efforts too. The […]

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