Author Archives: Patricia Inacio PhD

CRISPR Therapeutics and Vertex have launched two Phase 3 trials to assess the safety and effectiveness of CTX001, an experimental gene-editing cell therapy, one in children with sickle cell disease (SCD) and another for those with transfusion-dependent beta thalassemia (TDT). Both Phase 3 studies will enroll up to…

Robotic ducks were delivered recently as comfort companions to children with sickle cell disease (SCD) at the Children’s Regional Hospital at Cooper in South Jersey. The life-sized cuddly robot, called “My Special Aflac Duck,” were made available free to children, 3 and older, in a new collaboration between…

Emmaus Life Sciences has launched a full-service telehealth platform that will allow people in the U.S. with sickle cell disease (SCD) greater and easier access to the approved therapy Endari (L-glutamine). Patients will be able to receive a same-day physician’s prescription for Endari, which can…

Note: This story was updated March 29, 2022, to remove a reference to Phi Beta Sigma as the oldest honor society in the nation. Phi Beta Kappa is the nation’s oldest honor society. The Sickle Cell Disease Association of America (SCDAA) and the Phi Beta Sigma Fraternity…

Sangamo Therapeutics is assuming all rights to SAR445136, an investigational gene-edited cell therapy co-developed with Sanofi and now in a Phase 1/2 trial in adults with severe sickle cell disease (SCD). Early trial data support the therapy’s safety and tolerability, and its effectiveness at raising levels of fetal hemoglobin in patients,…

Imara has announced that it will change the main goal of its ongoing Phase 2b Ardent trial, which is assessing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). The trial’s new main goal is the annual rate of vaso-occlusive crises (VOCs) —…

Scientists Kenneth Ataga, MD, and Santosh Saraf, MD, have received a $3.2 million grant to continue investigating the potential of machine learning as a method to help identify sickle cell disease (SCD) patients at high risk of developing progressive kidney disease. The grant awarded by the…

Health Canada has expanded its approval of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to patients who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. The decision comes about five months after the U.S. Food and Drug Administration (FDA) approved…

People with sickle cell disease (SCD) in Ghana — a country in sub-Saharan Africa, a region with the world’s highest SCD prevalence — often go undiagnosed until age 4 and during a pain crisis, a study into patients in Accra, the country’s capital, reported. These findings reflect the importance…

Two years since its establishment, the Networking California for Sickle Cell Care initiative has seen increasing numbers of specialized clinics and healthcare professionals knowledgeable about sickle cell disease (SCD) and is setting new goals to improve the lives of patients in the state, the Network announced in its…