Tag Archives: sickle cell disease

Woman in coma ‘evacuated’ from home

Julie Iswan, 43, who had sickle cell disease, was evacuated from her home. (SWNS) One woman said she was removed from her home while in a coma, and when she got out of intensive care, all her belongings were gone. Juliet Iswan, 43, was in Bristol Royal Infirmary last February after suffering a stroke that… Read More »

Experimental gene therapy allows children with hereditary deafness to hear

A small study Published on Wednesday documents significantly restored hearing in five out of six children treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar developments in an 11-year-old boy being treated there. And earlier this month Chinese researchers published A study showing the same thing in two other children. So far,… Read More »

Experimental gene therapy allows children with hereditary deafness to hear

Gene therapy has allowed many children born with hereditary deafness to hear. A small study published Wednesday documented significant improvement in hearing in five out of six children treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar developments in an 11-year-old boy being treated there. And earlier this month, Chinese researchers published… Read More »

Experimental gene therapy allows children with hereditary deafness to hear

Gene therapy has allowed many children born with hereditary deafness to hear. A small study published Wednesday documented significant improvement in hearing in five out of six children treated in China. On Tuesday, the Children’s Hospital of Philadelphia announced similar developments in an 11-year-old boy being treated there. And earlier this month, Chinese researchers published… Read More »

Why are genetically modified fish being bred to fight rare childhood diseases?

Scientists have genetically engineered a new species of zebrafish with transparent skin to allow full observation of their internal organs – Richard White/Boston Children’s Hospital At the heart of Doha’s Education City, a vast, sun-bleached campus of tech companies and universities, lies a healthcare facility that Qatar hopes will lead the way in personalized medicine… Read More »

Promising new gene therapies for sickle cell remain unavailable in countries where they are needed most

Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for sickle cell disease, Dongre says he’s “praying for the cure to come to us.” But experts say a one-time treatment… Read More »

FDA approves 2 gene therapies for sickle cell. One of the first to use the CRISPR editing tool

Regulators approved two gene therapies for sickle cell disease on Friday. Doctors hope they may be able to cure painful, inherited blood disease that mostly affects black people in the US The Food and Drug Administration said the one-time treatments can be used for patients ages 12 and older with severe forms of the disease.… Read More »