Sandy Macrae, PhD, Chief Executive Officer of Sangamo Therapeutics, a company developing genetic therapies for a variety of rare conditions, including hemophilia A, Fabry disease, beta-thalassemia, sickle cell disease, mucopolysaccharidosis I (MPS I) and MPS II, talks about the challenges of recruiting patients for clinical trials.

Dr Macrae said, “These are very small numbers of patients and to do the trial well, you need to exclude certain medical conditions and patients with the mildest or the most severe disease,” adding, “so you need to take the time to recruit patients.”

At the same time, Dr. Macrae acknowledged that the goals of patients are different from those of regulators and pharmaceutical companies. For example, patients do not want to be the first person in an early stage clinical trial nor do they want to be in the placebo group.  Therefore, “It really is a matter of slow deliberate conversations with the patients and the community to make sure we do the right thing,” noted Dr. Macrae.

For more information about Sangamo and their clinical trials, visit

To learn more about ways to talk to patients about clinical trials, visit our CME/CNE program on the subject at


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