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Children's Hospital Colorado helps shape national standards for neurodevelopmental and neurocognitive care in sickle cell disease

New national standards recommend earlier screening and lifelong monitoring for development and cognition in individuals with SCD, aiming to reduce variability in care. The guidance, developed in collaboration with Children's Colorado, provides a tiered approach to care, including surveillance and evaluation.

SourceChildren's Hospital Colorado·JournalPediatric Blood & Cancer·DateJul 28, 2026

Study finds regional differences in sickle cell disease in New York state

A new study reveals significant variation in sickle cell disease hospitalizations and costs across New York State, with regional differences in access to specialized care and treatment impacting outcomes. Long Island and New York City show high costs and complex cases, while Central New York has the longest average length of stay.

SourceNew York University·JournalJAMA Network Open·DateMay 1, 2026

Gene editing therapy shows success against severe sickle cell disease

A gene-edited treatment has shown remarkable success against severe sickle cell disease, with 27 out of 28 patients achieving a functional cure and no painful crises. The therapy uses CRISPR/Cas12a technology to modify stem cells and increase levels of fetal hemoglobin.

SourceCleveland Clinic·JournalNew England Journal of Medicine·DateApr 1, 2026
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Periods may trigger pain for many who have sickle cell disease

A nationwide study found that periods can trigger severe pain in many women and girls with sickle cell disease, leading to emergency department visits and hospitalizations. The study's authors recommend a routine menstrual history screening as part of annual sickle cell care and connecting patients with reproductive health specialists.

SourceUniversity of California - San Francisco·JournalJAMA Network Open·TypeSurvey·DateFeb 19, 2026

New study: high efficiency of severe thalassemia prevention with HTS based carrier screening

A recent study found that strict adherence to High-Throughput Sequencing (HTS) technology based carrier screening can achieve high efficiency in preventing severe thalassemia birth defects. The study identified 15.07% of women as carriers of thalassemia and confirmed 59 fetuses with severe thalassemia, all of which were in high-risk co...

SourceBGI Genomics·JournalJournal of Genetics and Genomics·DateMay 8, 2025

CHOP and Penn Medicine researchers develop “in vivo” RNA-based gene editing model for blood disorders

CHOP and Penn Medicine researchers have developed a proof-of-concept model for delivering gene editing tools directly into diseased blood cells within the body. This approach aims to reduce costs and increase access to gene therapies for blood disorders, which currently require chemotherapy and stem cell transplants.

SourceChildren's Hospital of Philadelphia·JournalScience·DateJul 27, 2023
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In utero exposure to extreme morning sickness may cause developmental deficits in children

A new study from UCLA has found that women experiencing extreme morning sickness during pregnancy are three times more likely to have children with attention disorders and language delays. In utero exposure to HG may be linked to nutrient deficiency and life-long effects on the exposed fetus.

SourceUniversity of California - Los Angeles Health Sciences·JournalEuropean Journal of Obstetrics & Gynecology and Reproductive Biology·DateApr 20, 2015