Research

Over the past year, important contributions have been made in sickle cell research. This progress are critical steps towards finding new treatments and a universal cure for sickle cell conditions.

The Hibiscus Study Seeks Volunteers

Medicaid and CHIP Sickle Cell Disease Report, T-MSIS Analytic Files (TAF) 2017

CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine

Novartis Investigational Therapy Crizanlizumab (SEG101) Receives FDA Breakthrough Therapy Designation for the Prevention of Vaso-occlusive Crises in Sickle Cell Disease.

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease.

GBT Announces the FDA Agrees to Submit a New Drug Application for the Approval of Voxelotor.

FDA Approves Hydroxyurea Tablets for Pediatric Sickle Cell Anemia

Imara Doses First Patient in Phase 2a Clinical Trial of IMR-687 for Sickle Cell Disease.

Emmaus Life Sciences Announces Availability of Endari™ (L-glutamine oral powder) for Sickle Cell Disease in the United States.

U.S. Food and Drug Administration granted regular approval to hydroxyurea to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients from 2 years of age and older with sickle cell anemia with recurrent moderate to severe painful crises.

The Hibiscus Study Seeks Volunteers

Medicaid and CHIP Sickle Cell Disease Report, T-MSIS Analytic Files (TAF) 2017

CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine

Novartis Investigational Therapy Crizanlizumab (SEG101) Receives FDA Breakthrough Therapy Designation for the Prevention of Vaso-occlusive Crises in Sickle Cell Disease.

CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease.

GBT Announces the FDA Agrees to Submit a New Drug Application for the Approval of Voxelotor.

FDA Approves Hydroxyurea Tablets for Pediatric Sickle Cell Anemia

Imara Doses First Patient in Phase 2a Clinical Trial of IMR-687 for Sickle Cell Disease.

Emmaus Life Sciences Announces Availability of Endari™ (L-glutamine oral powder) for Sickle Cell Disease in the United States.

U.S. Food and Drug Administration granted regular approval to hydroxyurea to reduce the frequency of painful crises and the need for blood transfusions in pediatric patients from 2 years of age and older with sickle cell anemia with recurrent moderate to severe painful crises.

Sickle Cell News