Clinical trials in rare diseases are complex and present unique challenges. However, patients in these trials are very vocal, with caretakers, family members, and advocates eager to share their experiences. By using actionable strategies to find, listen to, and learn from patients themselves, researchers can include their perspective in study design.
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In Part Three of our Decoding FDA Gene Therapy Guidances series, we discuss the need for thorough long-term follow-up studies for these products, as…
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