Rising Incidence and Newborn Screening Programs Spur Rapid Growth in Sickle Cell Anemia Diagnostics
Asia Pacific Leads Next Wave of Sickle Cell Screening Adoption as Public Health Funding Expands Global Sickle Cell Anemia ...
Medical Xpress29d
Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
Their discovery may chart a path to a small-molecule drug that could be made available to all patients with sickle cell disease ... that raises fetal hemoglobin levels. The FDA approved it ...
sicklecellanemianews9d
Newborn sickle cell screening program aids outcomes for children
More than 500,000 babies were tested for sickle cell disease in Catalonia’s newborn screening program, with 160 cases ...
soapcentral3d
What is the Sickle Cell Disease from The Pitt? Explained
In The Pitt Season 1 Episode 2, sickle cell disease (SCD) is the main focus, as it brings to light the tough medical and racial challenges that people with this illness have to face every day.
Fred Hutch29d
Towards accessible gene therapy: in vivo base editing to cure Sickle Cell Disease
Sickle cell disease (SCD ... This can result in severe pain and anemia for people living with the disease. SCD is caused by a mutation in a single nucleotide of the hemoglobin gene. So far, the only ...
Business Insider29d
European Commission Adopts Positive Decision for Orphan Medicinal Product Designation of Agios’ Mitapivat in Sickle Cell Disease
The primary endpoints were hemoglobin response ... and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated anemia and phenylketonuria ...
Hosted on MSN28d
Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
Their discovery may chart a path to a small-molecule ... with sickle cell disease, as well as those with beta thalassemia. In sickle cell disease, a gene mutation causes the hemoglobin molecules ...
Stockhouse29d
European Commission Adopts Positive Decision for Orphan Medicinal Product Designation of Agios' Mitapivat in Sickle Cell Disease
The primary endpoints were hemoglobin response ... medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated ...