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Building the patient community

Gene Therapy volume 24, pages 547–550 (2017)Cite this article

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Abstract

There are many challenges in conducting rare disease research. The conditions are often poorly understood, small patient populations are dispersed around the world, and there are limited funding opportunities. Patient groups can serve as a key partner in overcoming these challenges, as they understand the impact of rare conditions on patients’ lives. This gives patient groups valuable scientific insights into the disease. This can be used to create research strategies, address research bottlenecks and directly fund research that appropriately addresses patient needs. Patient groups can also play a critical role in recruiting and retaining patients for clinical trials, which reduces time and resource waste. By partnering with patient groups, research teams can improve efficiency of research and best meet the needs of patients. Researchers can also play an important role in building and supporting patient groups to unlock these benefits.

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Author information

Authors and Affiliations

  1. Findacure Foundation, Cambridge, UK

    F Raffai

  2. AKU Society, Cambridge, UK

    O Timmis

Authors
  1. F Raffai
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  2. O Timmis
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Correspondence to F Raffai.

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Competing interests

FR is the Executive Director of Findacure and a trustee of the Cambridge Rare Disease Network. OT is the CEO of the AKU Society. The authors have no other relevant affiliations or financial involvement with any organisation or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

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Raffai, F., Timmis, O. Building the patient community. Gene Ther 24, 547–550 (2017). https://doi.org/10.1038/gt.2017.33

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