A few weeks ago, I attended a sickle cell awareness event for pharmacists. I was simply there as an interested sickle cell disease patient; I don’t work in the pharmaceutical industry. One major topic of discussion was the lack of progress toward cures for sickle cell disease.

Research is essential for change. For example, researching different diets and exercise regimens has helped me learn how to better manage my sickle cell disease. I’m open to discovering new techniques because no one drug or treatment will work for everyone. I’m also keen to learn about…

I work in the research and development field, an industry I didn’t know much about until I completed my first college degree. In this field, I’ve developed a vast understanding of how clinical trials work and why they are necessary to develop novel treatments for various illnesses and diseases.

IMR-687 (tovinontrine), an experimental oral therapy for sickle cell disease (SCD), reduced the rate of painful vaso-occlusive crisis (VOC), lowered hospitalization due to VOCs, and extended the time to a first VOC in SCD patients, according to new data from an open-label extension of a Phase 2 clinical trial.

Several clinical research sites seeking to find new treatments for people with sickle cell disease (SCD) are joining a collaborative network created by the American Society of Hematology Research Collaborative (ASH RC). The initiative, called ASH RC Sickle Cell Disease Clinical Trials Network, has already engaged about 24,000 patients…

Imara has completed patient enrollment for its Phase 2b Ardent clinical trial, which is testing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). “We are excited to have enrolled [participants] from across the world, including in Africa, making this a truly global…

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…

Global Blood Therapeutics’ lead therapeutic candidate voxelotor can induce rapid, robust, and sustained improvements in the amount of hemoglobin in adolescents and adults with sickle cell disease (SCD), according to interim results from the first part of a Phase 3 trial. The latest results of GBT’s clinical…

Modus Therapeutics is going to launch a Phase 1 clinical study in the United States to evaluate the potential of its lead candidate sevuparin for the treatment of sickle cell disease (SCD). In the trial, the company will broaden the clinical scope and usefulness of sevuparin to treat…

Once-daily treatment with voxelotor, Global Blood Therapeutics’ lead therapy candidate, can effectively increase the amount of hemoglobin in about 58% of patients with sickle cell disease (SCD) in just 12 weeks, according to results from Part A of a Phase 3 trial. Supported by these positive results, the company…