Rare diseases are rapidly becoming a high priority therapeutic area for our industry. This is needed, especially when fewer than 5% of approx 7,000 rare diseases have drug therapies available, and many of these provide limited benefit. There is also a business case to this, due to a supportive reimbursement environment, particularly in the US. […]

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Rare disease drug development is a rapidly expanding field with growing pressure for getting more orphan drug approved to treat rare diseases. There is a vast consensus among patients, health care bodies, governments etc. to expedite drug development in this field. Some wonderful numbers that attest to growing interest in rare diseases and orphan drugs […]

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