In Europe, approximately 250 people ages 6 and older have one of 8 additional gating mutations- KALYDECO is the first medicine to treat the underlying …
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[[Whitepaper ]] Disruptive Innovation in Rare Disease Markets
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Choosing the Right Channel Strategy for Specialty Product Success
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[Whitepaper] Challenging Conventional Patient Recruitment and Care Strategies for Rare Disease Studies
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[[whitepaper]] The New Reality: Maximizing Value in Rare Disease Launches
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What’s New in Rare Cancer Research? (Spoiler Alert: A Lot!)
