BACKGROUND: Young people’s experiences of healthcare as they move into adult services can have a major impact on their health, and the transition period for young people with sickle cell disease (SCD) needs improvement. In this study, we explore how young people with SCD experience healthcare during this period of transition. METHODS: We conducted a …
Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older. “Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million globally, who live with this …
Sickle cell disease (SCD) is one of the most prevalent genetic conditions in sub-Saharan Africa. It is a chronic, lifelong disease often characterized by severe pain. However, SCD has received little investment terms of health research, though there is currently a growing pool of SCD data from health and research facilities in different countries. To …
Victoria Gray slides open a closet door, pulls out a suitcase and starts packing piles of clothes. “My goodness,” says Gray. “Did I really bring all this?” Gray, who has sickle cell disease, is the first patient with a genetic disorder whom doctors in the United States have tried to treat using the powerful gene-editing …
Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates Foundation will …
The clinical presentation of acute chest syndrome is similar whether due to infectious or non-infectious causes, thus antibiotics are usually prescribed to treat all episodes. Many different pathogens, including bacteria, have been implicated as causative agents of acute chest syndrome. There is no standardized approach to antibiotic therapy and treatment is likely to vary from …
BACKGROUND: Newborn screening provides early diagnosis for children with sickle cell disease (SCD), reducing disease-related mortality. We hypothesized that rapid point-of-care (POC) Sickle SCAN would be reliable in Haiti and would assist newborn screening. METHODS: Dried blood specimens were obtained from infant heel sticks and analyzed by isoelectric focusing (IEF) at a public hospital in …
This past weekend, Milwaukee hosted a film festival that was the first of its kind not only in the city, but in the United States. The Minority Health Film Festival, which ran Sept. 12-15, was the country’s first film festival created to address the topic, said Heidi Moore, director of emerging markets and inclusion at …
It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR, making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness. …
The first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a genetic disorder. “It is just amazing how far things have come,” says Victoria Gray, 34, of Forest, Miss. “It is wonderful,” she told NPR in an exclusive interview after undergoing the landmark treatment …
