News

The Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease (SCD).…

The Food and Drug Administration (FDA) has expanded the approval of Casgevy® (exagamglogene autotemcel) to include patients aged 2 years and older with either…

The FDA has granted supplemental approval to exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals) for patients 2 years and older with sickle cell disease (SCD…

El Sabah Children’s Hospital has recorded over 300 cases of sickle cell disease in two years without diagnostic facilities, according to the Executive Director, Dr…

The spleen is one of the first organs injured in sickle cell disease (SCD) [1]. Splenic impairment in SCD children can silently progress toward functional asplenia…

WASHINGTON — The Food and Drug Administration Wednesday granted supplemental approval for a breakthrough CRISPR gene therapy medication, making it the first…

AbstractBackground and objectivesSickle cell disease is a major haemoglobin disorder, and foetal haemoglobin (HbF) remains the strongest modifier of disease…

AbstractImportance: Suboptimal transitions from pediatric to adult health care can negatively impact disease outcomes, medication regimen adherence, and disease…

Sickle cell disease (SCD) is the most common inherited clinically significant blood disorder, affecting at least 100 000 Americans and millions around the world.…

The approval expands the use of Casgevy to patients as young as 2 years old for the treatment of sickle cell disease and| beta thalassemiaThe USFDA issued a…