Search Results for: Cáceres

Putting patients first in the translational research pipeline

This is part II of a two-part blog series recapping the 2018 BIO International Convention. Read part I: Forecasting the convergence of artificial intelligence and precision medicine. The hope to improve people’s lives is what drives many members of industry and academia to bring new products and therapies to market. At the BIO International Convention…

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A double-shot of good news for SCID: Promising transplant and gene therapy data

In the world of fatal congenital immunodeficiency diseases, good news is always welcome, because most patients die before their first birthday if not treated. Babies with severe combined immunodeficiency disease, aka SCID or the “bubble boy disease,” now have more hope for survival thanks to two pieces of good news. Transplants are looking up First…

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Gene therapy: Two years and holding

For the Cáceres family of Argentina, it’s a joyous holiday homecoming. Agustín, who received gene therapy at 5½ months of age, journeyed with his family to Boston for a check-up and got a clean bill of health. Agustín was born with the rare immune-deficiency disorder SCID-X1. More popularly known as “bubble boy” disease, it left…

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A new start for gene therapy for “bubble boy” disease: First U.S.-treated patient doing well

Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns. After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom….

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