Posts Tagged: bubble baby

News

California-backed cure for ‘bubble baby’ disease stalls — again

A pipette drops stem cell research fluid on a special container. (Photo: CI Photos, via Shutterstock)

The “bubble babies” saga and a California-financed cure for their life-threatening affliction has hit another snag, more than two years after a British company abandoned the effort. It is a story that involves more than $40 million from California’s stem cell agency, federal regulators, the University of California, the agonizingly slow pace of science and 20 children who have been denied care — not to mention a company called Orchard Therapeutics PLC.

News

‘Bubble baby’ treatment shifted to stem cell agency, UCLA

Evangelina Padilla-Vaccaro’s medical team gathers around her on the day she received her gene-therapy stem-cell transplant. (Photo: Padilla-Vaccaro family, via UCLA Health)

A London-based biotech firm has given up its life-saving treatment for the bubble baby disease and turned it over to California’s $12 billion stem cell agency and UCLA, where it was developed with tens of millions of taxpayer dollars.

News

Frustration, anxiety mark families’ hopes for ‘bubble baby’ cure

Monica Nava and her 1-year-old daughter Clementine, who suffers from the "bubble baby" affliction. (Family photo).

The story about Jakob, Sheersha  and Clementine is a 6,000-mile biomedical tale that spans the Atlantic. The story ranges from the Saskatchewan River in Canada to the dusty Tehachapi mountains in drought-plagued Southern California. And it is a story of children with a terrible and rare genetic affliction known as the bubble baby disease.

News

Emotions soar at stem cell board meeting

Throat cancer patient Karl Trede, who was treated with the "eat me" protein therapy. (Photo: California Institute for Regenerative Medicine)

In an emotion-choked session, the mother of a six-year-old girl thanked California’s stem cell agency for saving the life of her daughter. “Thank you for keeping my family complete,” said Alysia Padilla-Vaccaro, her voice cracking as she spoke to the governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

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