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. Author manuscript; available in PMC: 2011 Jul 13.
Published in final edited form as: Mol Genet Metab. 2008 Nov 13;96(1):20–26. doi: 10.1016/j.ymgme.2008.10.003

Table 3.

Skill Sets Essential for Research on Rare Diseases

  • Recruiting and retaining subjects

  • Developing multiple streams of extramural research funding

  • Ability to achieve disease-centered development/philanthropy

  • Developing therapeutic agents with small “markets”

  • Obtaining/gaining access to therapeutic agents

  • Establishing equipoise in investigators and patients