A $12.2-million grant awarded to the New York-based Mount Sinai Health System will enable a nationwide observational study to help better personalize sickle cell disease (SCD) treatments. “Sickle cell traditionally has been a neglected disease, but it benefited from a flurry of innovation over the last decade and…
The number of red blood cells that produce the abnormal hemoglobin S (HbS), which characterizes sickle cell disease (SCD), is reduced by the experimental gene therapy BCH-BB694, as is their tendency to take a sickle-like shape. That’s according to single-cell data from seven patients in an ongoing Phase…
A portrait of 4-year-old Kynnedi Sturges, who is living with sickle cell disease (SCD) in Camden, Arkansas, was unveiled last week in an international art exhibit by the nonprofit Beyond the Diagnosis. Displaying artworks of 40 children with rare diseases, the exhibit is being hosted by…
The Georgia State University Research Foundation has received a grant from the Centers for Disease Control and Prevention (CDC) to support the ongoing Sickle Cell Data Collection (SCDC) program in that state. Established by the CDC in 2015, the program seeks to assess the frequency of…
To help heighten awareness of sickle cell disease (SCD) and pediatric cancers, while bringing comfort and relief to patients, the supplemental insurance provider Aflac recently gifted its My Special Aflac Duck social robot to children at the University of New Mexico (UNM) Children’s Hospital in Albuquerque. The delivery…
Rates of life-threatening infections by pneumococcal bacteria have significantly declined, by about 80%, among children with sickle cell disease (SCD) since the introduction of pneumococcal conjugate vaccines 20 years ago. That’s according to a study analyzing more than two decades of healthcare data from young SCD patients in metropolitan…
A $1.9-million federal grant has been awarded to a clinical trial that seeks to find effective ways to improve adherence to hydroxyurea treatment among children and young adults with sickle cell disease (SCD). Marketed as Droxia and Siklos, hydroxyurea is an oral therapy approved to reduce the frequency…
A scientist whose discoveries paved the way for the development of an investigational gene therapy for sickle cell disease (SCD) and other blood disorders has been awarded the third Elaine Redding Brinster Prize in Science or Medicine. Stuart Orkin, MD, received the award — given annually to a researcher…
Because many individuals of African ancestry don’t know they have the sickle cell trait or what it means to be a carrier, 23andMe and the nonprofit organization Sickle Cell 101 are collaborating to expand an awareness program. With the collaboration, up to 1,000 U.S. adults of African…
Treatment with the investigational gene-editing therapy OTQ923 led to the sustained production of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three adults with severe sickle cell disease (SCD), according to data from a Phase 1 study. The treatment involved collecting red blood cell precursors, called hematopoietic stem…
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