Prompted by the disparities in care between children and adults with sickle cell disease (SCD) and inspired to raise awareness about rare diseases, Cory Lewis has made it his personal mission to improve health for the African-American sickle cell community. Lewis, who is a 27-year-old leasing agent and has…
News
GBT601 and inclacumab, experimental therapies from Global Blood Therapeutics (GBT) for sickle cell disease (SCD) and associated vaso-occlusive crises (VOCs), were generally safe and showed signs of biological activity in healthy volunteers, according to data from Phase 1 clinical trials. These findings will be presented by GBT through two…
Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults with sickle cell disease (SCD). That’s according to nearly three years of data from the Phase 3 HOPE…
Through a new partnership between Emmaus Life Sciences and UpScript, patients with sickle cell disease (SCD) will soon be able to consult with a physician from home and have greater access to Endari (L-glutamine). Through telehealth — using computers and mobile devices to manage and receive…
The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…
The U.S. Food and Drug Administration (FDA) has cleared Beam Therapeutics to start clinical testing of BEAM-101, the company’s investigational gene editing cell therapy for sickle cell disease (SCD). “BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables…
Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…
Oxbryta (voxelotor), Global Blood Therapeutics’ first-in-class oral therapy for sickle cell disease (SCD), has won the 2021 Prix Galien USA Award for best biotechnology product. The award is among the health industry’s most prestigious honors, and recognizes an outstanding product that has improved patient well-being and impacted human…
In support of its work to develop a rapid test that can be used to diagnose sickle cell disease (SCD), a team of undergraduate researchers at Lehigh University has received a $15,000 award as part of the National Institutes of Health’s Healthcare Technologies for Low-Resource Settings Prize. “I was…
A point-of-care, low-cost device called Gazelle — that’s both portable and easy to use — can accurately diagnose sickle cell disease (SCD) in less than 15 minutes, according to a study done in India. As noted by researchers, this device has the potential to be adopted as a…
Recent Posts
- Agios to seek accelerated approval in US of oral mitapivat for SCD
- Japanese agency awards $32M to advance sickle cell treatment to trials
- Intentional lifestyle changes got me through winter without a crisis
- Chicago nurses lead charge to speed up SCD emergency care: Study
- Recognizing the warning signs and symptoms of a sickle cell crisis
- US senators seek 5 more years of federal funding for stem cell program
- Adults with SCD face high risk of stroke in their 30s and 40s, study finds
- Sickle cell doctors face higher burnout than other specialists: Study
- Disease burden heavy for SCD patients in US, despite promise of gene therapy
- The conversation we avoid: Telling children they have sickle cell disease