Sickle Cell Disease news and research updates
Research readouts, treatment and access decisions, and community updates for people living with sickle cell disease and the people who care for them — each one dated, with a link to the original source where there is one.
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FDA Expands Casgevy Approval to Children as Young as 2 Years With Sickle Cell Disease or Transfusion-Dependent β-Thalassemia
The FDA has granted supplemental approval to exagamglogene autotemcel (Casgevy; Vertex Pharmaceuticals) for patients 2 years and older with sickle cell disease (SCD) with…
El Sabah hospital records 300 sickle cell cases, lacks diagnostics
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. Justin Burono…
Subtotal Versus Total Splenectomy in Children With Sickle Cell Disease: Clinical Outcomes and Splenic Function Assessed by Pocked Red Blood Cell Count
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, exposing…
FDA Approves First Gene Therapy Technology to Treat Children With Sickle Cell Disease
WASHINGTON — The Food and Drug Administration Wednesday granted supplemental approval for a breakthrough CRISPR gene therapy medication, making it the first available to treat…
Sickle Cell Disease: World’s highest burden
•Why place of birth shouldn’t decide child’s fate For thousands of Nigerian families, the joy of welcoming a new born is often overshadowed by an invisible threat; Sickle Cell…
Efficacy of curcumin and resveratrol in the synergistic activation of foetal haemoglobin in sickle cell disease
Abstract Background and objectives Sickle cell disease is a major haemoglobin disorder, and foetal haemoglobin (HbF) remains the strongest modifier of disease severity…
Coping Strategies and Social Support for Transition Readiness Among Youth With Sickle Cell Disease
Abstract Importance: Suboptimal transitions from pediatric to adult health care can negatively impact disease outcomes, medication regimen adherence, and disease self-management…
Evaluating high-sensitivity troponin levels in people with sickle cell disease in the emergency department
Sickle cell disease (SCD) is the most common inherited clinically significant blood disorder, affecting at least 100 000 Americans and millions around the world. Red blood cells…
FDA Expands Casgevy for Children 2+ with Sickle Cell Disease
The approval expands the use of Casgevy to patients as young as 2 years old for the treatment of sickle cell disease and| beta thalassemia The USFDA issued a supplemental approval…
FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease
The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell…
Vertex Announces US FDA Approval for Expanded Use of CASGEVY® for the Treatment of People Ages 2 Years and Older With Sickle Cell Disease or Transfusion-Dependent Beta Thalassemia
First and only approved genetic therapy to treat children as young as 2 years for both severe sickle cell disease and transfusion-dependent beta thalassemia - - Approximately…
Curcumin-based gel may ease sickle cell pain, early data suggest
A low dose of Vascarta ‘s VAS-101, a curcumin-based gel being developed for sickle cell disease (SCD), was well tolerated and showed early signs of reducing red blood cell…