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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U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease
CAMBRIDGE, Mass. , July 07, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative…
Gene Therapy Casgevy Approved for Younger SCD and TDT Patients
The Food and Drug Administration (FDA) has expanded the approval of Casgevy ® (exagamglogene autotemcel) to include patients aged 2 years and older with either sickle cell disease…
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…
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…
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…
Sickle cell epidemic
June 19 is the annual date the world marks ‘International Sickle Cell Day’. The theme for this year’s own is, “Closing the Survival Gap: Equity in Sickle Cell Care”. Sickle Cell…
23 year old becomes first person in Louisiana to be functionally cured of sickle cell disease
Daniel Cressy became the first person in the Gulf South to be functionally cured of sickle cell disease through gene editing. Calling it his "second birthday," he said the…