SCD

Disease

Related News

Transformative Therapies Advance Treatment for Sickle Cell Disease

Apr 02, 2026

Transformative therapies including stem cell transplant and gene therapy offer sickle cell disease patients potential freedom from the SCD phenotype with reduced morbidities. About 90% of patients successfully switch to donor blood cell production after transplant, while gene therapies work by editing the patient's own stem cells. These treatments require careful consideration of disease severity and potential risks including major adverse events.

Sanofi's Rilzabrutinib Gains Regulatory Designations in Japan and U.S. for Rare Diseases

Mar 03, 2026

Sanofi's oral BTK inhibitor rilzabrutinib received orphan drug designation in Japan for IgG4-related disease and warm autoimmune hemolytic anemia, while also securing U.S. FDA breakthrough therapy designation for the rare anemia indication.

Gene Therapy for Sickle Cell Disease Remains Unaffordable in Africa Despite US, UK Approvals

Feb 26, 2026

Approved gene-editing therapies for sickle cell disease priced at $2.2 million remain out of reach in Africa, where 80% of the world's cases occur, as Uganda launches mandatory newborn screening.

Related Clinical Trials

NCT ID	Title	Status	Phase
NCT07188766	Efficacy of Liberal Versus Restricted IV Fluid Approach in the Management of Sickle Cell Vaso-Occlusive Crisis	RECRUITING	NA
NCT07009821	Neonatal Screening for Haemoglobinopathies EmoCamp	NOT_YET_RECRUITING
NCT06962501	Acute Oral Ketone Monoester Supplementation and Resting-state Brain Connectivity	COMPLETED	NA
NCT06746909	Identification of Neuroinflammation and Neuroimaging Biomarkers Through Data Driven Artificial Intelligence Techniques for Unraveling the Heterogeneity of Aged Subjects at Risk of Dementia and to Better Inform Prevention Strategies	RECRUITING	NA
NCT06601894	The Relationship of Platelet Counts With Sickle Cell Anemia in the Eastern Region of Saudi Arabia	NOT_YET_RECRUITING
NCT06335836	The Effects of Social Isolation and Social Interaction on the Risk of Dementia Progression and Brain Function in SCD (Subjective Cognitive Decline, SCD)	RECRUITING
NCT06122415	The Swedish BioFINDER - Memory Clinic Study	RECRUITING
NCT06120361	The Swedish BioFINDER - Primary Care Study	RECRUITING
NCT05952817	Effect of Virtual Reality on Pain and Function in Children With Sickle Cell Disease: A Randomized Controlled Trial	COMPLETED	NA
NCT05682469	Effects and Mechanism of the Fully Immersive Virtual Reality Cognitive Training Based on Leisure Activities	UNKNOWN	NA