UCSF Benioff Children's Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe anemia, with a need for lifelong ...
Aug 31, 2023
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Newborn screening for cystic fibrosis (CF) was fully implemented in all 50 states in the U.S. by 2010, but delays in timeliness of evaluation for infants with positive newborn screen tests persist. Through evaluation of national ...
Jul 31, 2023
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Reducing the burden of sickle cell disease (SCD) requires substantial financial and political commitment to improving dataccollection, diagnosis, treatment and training. Doing so will positively impact the lives of millions ...
Jul 13, 2023
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Introducing widespread screening of newborns for a deadly disease called severe combined immunodeficiency, or SCID, followed by early treatment boosted the five-year survival rate of children with the disorder from 73% before ...
Jun 21, 2023
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Babies born in U.S. hospitals currently undergo routine newborn screening, a laboratory test to identify the risk of up to 60 treatable conditions. But hundreds of genetic disorders, including a growing number of devastating ...
May 8, 2023
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While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps. A new study published in Blood Advances finds that the average ...
Mar 16, 2023
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Scientists have developed a test that could greatly improve quality of life for infants with homocystinuria (HCU), a congenital disease that—if not treated early—causes serious complications. Research demonstrating the ...
Mar 15, 2023
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Minnesota is the first U.S. state to universally test newborns for cytomegalovirus, an easily transmissible virus and a leading cause of infant hearing loss and congenital birth defects.
Feb 9, 2023
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Babies undergo a newborn screening test for inherited metabolic disorders within seven days of birth. The test checks for risk factors such as hypothyroidism, phenylketonuria and maple syrup urine disease, which can lead ...
Jan 19, 2023
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Newborn screening (NBS) for spinal muscular atrophy (SMA), when combined with early treatment, results in better movement ability in affected children, including the ability to walk, when compared to children who are diagnosed ...
Jan 18, 2023
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