Hemophilia affects approximately 20,000 people in the United States.
Although scientists spend their days studying the inner workings of the disease, what moves them most is hearing from patients and their families about how uncontrolled bleeding in the joints impacted their or their loved one’s life – limiting mobility and causing pain and disability. The frequent infusions of clotting factors to minimize that damage takes a toll; from the time and effort they put into making sure they have the supply they need, to planning their lives around the treatments.
Fabry disease, also life threatening, affects as many as 1 in 7,000 people. Patients experience painful burning sensations in the extremities while an enzyme deficiency puts major organs at risk for permanent damage.
My role is to ensure we work closely with families and the community to understand the ways these diseases impact every aspect of a patient’s life, in hopes that the genomic treatments our scientists design have the chance to make a lifelong transformation for patients and their loved ones.
The scientists at Sangamo have invested years studying genomic medicine – a still-emerging field that has the potential to transform the treatment of several diseases. My job is, to work with our teams to help them get to know real patients so that we comprehend not just their diseases in medical terms, but the reality of their lives in personal terms. Connecting with people who live with these conditions fosters insight that shapes the creation of medicines, and the design and planning of clinical trials – ensuring those programs measure the effect on disease symptoms, as well as everyday quality of life.
Motivated to make a difference
The empathy cultivated by increased connection to patients and a deeper understanding of their experience is what I believe drives our scientists to make educated choices – and more thoughtful ones. The emotional insight into patients is critical in guiding every step of development. Connecting with patients and caregivers helps us ensure we are using the best genomic medicine approach to address their condition, as well as designing clinical trial protocols and defining endpoints that reflect the treatment goals that are important to patients.
When patients enter our lives, they also take up permanent residence in our hearts and minds, changing our science just as a new treatment may change their experience. Meeting them alters how we think about drug development. It sharpens our approach to designing treatments. It intensifies our commitment to creating treatments that will make a real difference.
Throughout my career in patient advocacy, I have found that connecting with patients, caregivers and organizations is just one aspect of my job. The other equally important part of my job is ensuring we bring the voice, perspective and experience of patients into our company.
View “Understanding new discoveries in genomic medicine” video here.
By Christeen Moburg
As the Head of Patient Advocacy at Sangamo, Christeen Moburg leads the Company’s efforts to engage patients and patient advocacy organizations to ensure the patient voice is integrated in all aspects of development and business decisions. Over the course of her career, she has built partnerships and worked collaboratively with many advocacy groups on developing and implementing strategic patient advocacy initiatives.