A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasn’t right. But it wasn’t until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis – a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
Stories of Hope
The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Ayaan Isaacs was born in South Africa on March 4th, 2016 as a seemingly healthy baby. But only a few days in to life, he contracted a life-threatening liver infection. He thankfully survived, only to have the doctors discover a few weeks later that he had something much more troubling – a rare disease that … Continue reading The Story of a South African Bubble Boy and a Gene Therapy That Gave Him His Life Back
Advocating for Huntington’s Disease: Daniel Medina’s Journey
In honor of Huntington’s Disease (HD) Awareness Month, we’re featuring a guest blog by HD patient advocate Daniel Medina. Daniel became actively involved in the HD community when he learned that his younger brother was at risk for inheriting this devastating neurodegenerative disease. Since then he has been a champion for HD awareness by organizing … Continue reading Advocating for Huntington’s Disease: Daniel Medina’s Journey
Stem Cell Profiles in Courage: Karl’s Fight with Cancer
When I think of a pioneer I have an image in my head of people heading west across the Americans plains in the 18th century, riding in a covered wagon pulled by weary oxen. Karl Trede doesn’t fit that image at all. He is a trim, elegant man who has a ready smile and a … Continue reading Stem Cell Profiles in Courage: Karl’s Fight with Cancer
Stem Cells Profile in Courage: Pat Furlong, Patient Advocate
One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary. So, over the next few weeks … Continue reading Stem Cells Profile in Courage: Pat Furlong, Patient Advocate
Don’t Sugar Coat it: A Patient’s Perspective on Type 1 Diabetes
“In the weeks leading up to my diagnosis, I remember making and drinking Kool-Aid at the rate of about a gallon per day, and getting up to pee and drink Kool-Aid several times a night. The exhaustion and constant thirst and the weight loss were pretty scary. Insulin saved my life, and it’s been saving … Continue reading Don’t Sugar Coat it: A Patient’s Perspective on Type 1 Diabetes
A patient perspective on how stem cells could give a second vision to the blind
October is Blindness Awareness month. In honor of the patients who suffer from diseases of blindness and of the scientists and doctors who work tirelessly to develop treatments and cures for these diseases, we are featuring an interview with Kristin Macdonald, a woman who is challenged by Retinitis Pigmentosa (RP). RP is a genetically inherited … Continue reading A patient perspective on how stem cells could give a second vision to the blind
A Patient Advocate’s Take on Sickle Cell Disease: The Pain and the Promise
September is National Sickle Cell Awareness Month. First officially recognized by the federal government in 1983, National Sickle Cell Awareness Month calls attention to sickle cell disease (SCD), a genetic disease that researchers estimate affects between 90,000 and 100,000 Americans. CIRM is funding a clinical trial focused on curing the disease with a stem cell-based … Continue reading A Patient Advocate’s Take on Sickle Cell Disease: The Pain and the Promise
Out of the mouths, or in this case hearts, of babes comes a hopeful therapy for heart attack patients
Lessons learned from babies with heart failure could now help adults Inspiration can sometimes come from the most unexpected of places. For English researcher Stephen Westaby it came from seeing babies who had heart attacks bounce back and recover. It led Westaby to a new line of research that could offer hope to people who … Continue reading Out of the mouths, or in this case hearts, of babes comes a hopeful therapy for heart attack patients
Stories of Hope: Stroke
Six months after surviving a stroke, Sonia Olea wanted to die. Her right leg was weak, her right arm useless. She had trouble speaking and even small tasks were challenging. Just making a phone call was virtually impossible. One morning, she woke up with her arm pinned in an awkward, painful position. After finally repositioning … Continue reading Stories of Hope: Stroke
The Stem Cellar 