Joana Carvalho, PhD, managing science editor —

An advisory committee of the Sickle Cell Disease Association of America is recommending that people with sickle cell disease (SCD) be considered a “vulnerable” population during the reopening of the U.S. economy, and remain at home as much as possible. Patients should also continue taking their medications, and adopt…

APCER Life Sciences has released a report confirming the safety profile of Endari (L-glutamine), according to Emmaus Life Sciences, which developed the therapy. Endari is an oral therapy for sickle cell disease (SCD) approved by the U.S. Food…

Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan drug status for naproxcinod, a potential treatment for for sickle cell disease (SCD). Orphan drug designation is given to experimental treatments for rare diseases (those that affect…

People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face many challenges, including the financial burden posed by their healthcare expenses, low treatment adherence and high treatment discontinuation rates, according to a study based on pharmacy claims data. The study, “Treatment patterns and…

FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review (ICER). The report focused on the clinical benefits and cost-effectiveness of Novartis’ Adakveo (crizanlizumab), Global…

A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an  orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The FDA recently also designated ARU-1801 a potential…

FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical trial. Patient recruitment is open for the second part of the trial (NCT03815695). Information about trial locations,…

The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This designation means the company will be eligible to receive a priority review voucher…

Global Blood Therapeutics (GBT) and Syros Pharmaceuticals have teamed up to discover, develop, and market new therapies for sickle cell disease (SCD) and beta thalassemia. Under the collaboration agreement, Syros will be responsible for identifying new therapeutic targets and the discovery of drugs that stimulate the…

Sickle Cell Disease News brought you daily coverage of important discoveries, treatment developments, clinical trials, and other key events related to sickle cell disease (SCD) throughout 2019. As we look forward to bringing you more news in 2020, we would like to remind you of the 10 most-read…