Author Archives: Margarida Azevedo, MSc

The Sickle Cell Disease Association of America (SCDAA) is collaborating with Emmaus Life Sciences to increase patient education and awareness about the complications of sickle cell disease (SCD). The partners aim to improve national awareness so that information about the disease can reach broader audiences through online…

MaxCyte, a company specialized in cell-based medicines, announced it has entered in a research agreement with the National Heart, Lung and Blood Institute (NHLBI) to develop potential therapies for sickle cell disease (SCD). Researchers will use MaxCyte’s gene-correction platform, Flow Electroporation Technology, in their work. The platform is…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to olinciguat (IW-1701) as a potential treatment of sickle cell disease (SCD), Ironwood Pharmaceuticals announced. Evidence suggests that the symptoms of sickle cell disease may be associated with nitric oxide deficiency in the blood. Nitric oxide…

The Vodafone Americas Foundation recently announced the winners of its 10th annual Wireless Innovation Project (WIP), which awarded $600,000 in three-year grants to tackle critical issues facing the global community. The first-place prize of $300,000 was awarded to a point-of-care platform called SMART, for its remote diagnostic testing targeting…

The U.S. Food and Drug Administration placed a clinical hold on the investigational new drug (IND) application for CTX001, an experimental therapy for the treatment of sickle cell disease, according to a statement by CRISPR Therapeutics and Vertex Pharmaceuticals. The hold on the clinical development of CTX001 will…

A significant proportion of children with sickle cell disease (SCD) are not receiving key recommended vaccines on time or at all, according to a new study examining pneumococcal and meningococcal vaccinations among children in Michigan. The study, “Pneumococcal and Meningococcal Vaccination among Michigan Children with Sickle Cell Disease,”…

The U.S. Food and Drug Administration recently accepted an investigational new drug (IND) application submitted by Bioverativ and Sangamo Therapeutics for BIVV003, a gene-edited cell therapy candidate for the treatment of sickle cell disease. BIVV003 uses a nonviral approach that uses zinc finger nuclease (ZFN) gene-editing technology to…

Physicians who use stigmatizing language in their patients’ medical records could be affecting the care they receive for years to come, according to a new Johns Hopkins study. Titled “Do Words Matter? Stigmatizing Language and the Transmission of Bias in the Medical Record,” and published in the Journal…

Three programs focused on sickle cell disease (SCD) planned by St. Jude Children’s Research Hospital will benefit from a $1 million Legacy Grant awarded by The Links Foundation. The foundation, the philanthropic arm of The Links — one of the United States’ oldest and largest African-American women’s volunteer service…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…