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 Meet Vivian

Vivian received life-saving gene therapy at UCSF

Days after her birth, Vivian was diagnosed with spinal muscular atrophy, a life-threatening genetic disorder that leads to debilitating weakness and wasting in infants. Before the development of innovative gene therapy in 2016, this diagnosis meant that parents had a devastating choice to make: hook their child up to breathing and feeding machines around the clock or let them die.

Shortly after her diagnosis, Vivian received this groundbreaking gene therapy at UCSF – a national leader in genomics.

“Every time I see Vivian, I get goosebumps. I went into medicine to help people get better. But for a long time, I was a doctor who helped parents make decisions about the life and death of their child. With Vivian I was actually able to make a difference.”

-- Dr. Jonathan Strober, Director of the Neuromuscular Clinic at UCSF Benioff Children’s Hospitals

At almost a year and a half, Vivian is hitting milestones that were once out of reach for children with her diagnosis, including breathing on her own, eating everything in sight, and pulling herself up to standing.

For Vivian’s parents, it’s difficult to imagine that alternate universe, where gene therapy is still a dream and Dr. Strober has no choice but to tell them that their little girl won’t make it. 

Our Beneficiaries

 

 
 
 
 
 
 
 
George Mark Children's House (GMCH) in San Leandro, California, is the first free-standing pediatric palliative care house in the United States. Founded in 2004, GMCH provides the comfort of home with the medical care of a top-notch hospital. GMCH exists to serve families with children who are chronically ill with a life-limiting illness and are seeking family-centered medical care that emphasizes quality of life in a compassionate, supportive atmosphere where kids can be kids and families can be families.

 

 

UCSF Benioff Children's Hospital, Oakland's mission is to protect and advance the health and well-being of children through clinical care, teaching, and research. The hospital cares for more than 240,000 patients annually and welcomes all children, regardless of their race, ethnicity, or ability to pay. With values such as RESPECT, INTEGRITY, QUALITY, TEAMWORK, DIVERSITY, and STEWARDSHIP, their vision is to be a leader in children’s health.