Rare Disease Day in Sacramento, California How can a condition that affects 30 million Americans—one in ten people—be called rare? Yet rare diseases are defined as those affecting fewer than 200,000 people. There are about 7,000 such diseases, and fewer than five percent have a treatment. Last Wednesday, people across the U.S. gathered to call … Continue reading Breaking the isolation of rare diseases
SCID
Friday Roundup: A better kind of blood stem cell transplant; Encouraging news from spinal cord injury trial; Finding an “elusive” cell that could help diabetics
Cool Instagram image of the week: Chemo- and radiation-free blood stem cell transplant showing promise Bubble baby disease, also known as severe combined immunodeficiency (SCID), is an inherited disorder that leaves newborns without an effective immune system. Currently, the only approved treatment for SCID is a blood stem cell transplant, in which the patient’s defective … Continue reading Friday Roundup: A better kind of blood stem cell transplant; Encouraging news from spinal cord injury trial; Finding an “elusive” cell that could help diabetics
Using the AIDS virus to help children battling a deadly immune disorder
More than 35 million people around the world have been killed by HIV, the virus that causes AIDS. So, it’s hard to think that the same approach the virus uses to infect cells could also be used to help children battling a deadly immune system disorder. But that’s precisely what researchers at UC San Francisco … Continue reading Using the AIDS virus to help children battling a deadly immune disorder
Progress to a cure for Bubble Baby Disease
Evie, a former "bubble baby" enjoying life by playing inside a giant plastic bubble 'Bubble baby disease' will one day be a thing of the past. That’s a bold statement, but I say it with confidence because of the recent advancements in stem cell gene therapies that are curing infants of this life-threatening immune disease. … Continue reading Progress to a cure for Bubble Baby Disease
Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
During this second week of the Month of CIRM, we’ve been focusing on the people who are critical to accomplishing our mission to accelerate stem cell treatments to patients with unmet medical needs. These folks include researchers, like Clive Svendsen and his team at Cedars-Sinai Medical Center who are working tirelessly to develop a stem … Continue reading Saving Ronnie: Stem Cell & Gene Therapy for Fatal Bubble Baby Disease [Video]
Patient Advocate helps her son while helping others
A mother’s experience with severe combined immunodeficiency (SCID) is driving her work as a patient advocate on a CIRM-funded clinical trial—helping shape research that could transform treatment for children born with this rare, life-threatening immune disorder. A Personal Journey with SCID Anne Klein’s son, Everett, was diagnosed with SCID—often called “bubble baby disease”—shortly after birth through newborn … Continue reading Patient Advocate helps her son while helping others
Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
There were lots of CIRM mentions in the news this week. Here are two brief recaps written by Karen Ring to get you up to speed. A third story by Todd Dubnicoff summarizes an promising finding related to heart disease by researchers in Singapore. CIRM-funded “bubble baby” disease therapy gets special designation by UK. … Continue reading Stem cell stories that caught our eye: bubble baby therapy a go in UK, in-utero stem cell trial and novel heart disease target
CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
If you have read our 2016 Annual Report (and if you haven’t you should, it’s brilliant) or just seen the cover you’ll know that it features very prominently a young girl named Evie Padilla Vaccaro. Evie was born with Severe Combined Immunodeficiency or SCID – also known as “bubble baby disease”; we’ve written about it … Continue reading CIRM-funded life-saving stem cell therapy gets nod of approval from FDA
Bye Bye bubble baby disease: promising results from stem cell gene therapy trial for SCID
Evangelina Padilla-Vaccaro(Front cover of CIRM's 2016 Annual Report) You don’t need data to see that Evangelina Vaccaro’s experimental stem cell therapy cured her of a devastating, often fatal immune disease. A quick look at her photos or videos shows a happy, healthy five‑year‑old with a full life ahead. A look at the data But the … Continue reading Bye Bye bubble baby disease: promising results from stem cell gene therapy trial for SCID
Stem Cell Stories that Caught our Eye: stem cell insights into anorexia, Zika infection and bubble baby disease
Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. Stem cell model identifies new culprit for anorexia. Eating disorders like anorexia nervosa are often thought to be caused by psychological disturbances or societal pressure. … Continue reading Stem Cell Stories that Caught our Eye: stem cell insights into anorexia, Zika infection and bubble baby disease