Beam Therapeutics announced positive Phase 1/2 trial data for BEAM-101 in sickle cell disease, showing significant HbF increases and reduced VOCs. They also revealed a new conditioning platform, ESCAPE, aiming to reduce the toxicity associated with current treatments.
Fulcrum Therapeutics, Inc. (FULC) saw its stock plummet by over 60% after its phase III REACH study for losmapimod in treating facioscapulohumeral muscular dystrophy (FSHD) failed to meet its primary endpoint. While this news is a setback for the company, Fulcrum remains focused on its other pipeline candidate, pociredir, for sickle cell disease, which has garnered significant attention and potential.
The Supreme Court has directed the Indian government to decide within four weeks on including an advisory for fortified rice distribution, emphasizing the need for caution for individuals with thalassemia and sickle cell disease. This directive stems from a public interest litigation highlighting the potential risks associated with iron fortification for these specific medical conditions.
Dr Praveen Gupta, Principal Director & Chief of Neurology at Fortis Hospital, sheds light on the neurological complications associated with sickle cell disease (SCD). He discusses the primary neurological complications, neurodevelopmental challenges in children, and the importance of early intervention and support systems. Dr Gupta emphasizes the role of medical care and community support in improving the quality of life for individuals with SCD.
Sickle cell disease (SCD) is a severe blood disorder affecting millions worldwide, particularly in regions like sub-Saharan Africa and India. This article explores the causes, prevalence, symptoms, diagnosis, treatment, and global efforts to combat this debilitating disease.
