For most of his 39 years, Charles Hough lived with extreme pain caused by sickle cell disease, which dramatically reduced blood flow throughout his body. But thanks to the gene therapy he received in 2017, Hough is now symptom-free. “I feel like I have…
News
To help empower individuals living with sickle cell disease (SCD), biopharmaceutical company Emmaus Life Sciences has teamed up with national specialty pharmacy US Bioservices on a treatment adherence program for patients. Through a team of clinicians, the new opt-in enhanced nursing program offers patients information and real-time…
News
FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell
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…
The National Institutes of Health (NIH) has awarded a grant to researchers at Carnegie Mellon University’s College of Engineering to support their project to develop a non-invasive, non-toxic and non-addictive pain treatment for people with sickle cell disease (SCD). “There is an urgent unmet need to…
California’s legislature has committed $15 million toward sweeping enhancement of adult sickle cell disease (SCD) care in a state with historically underserved patients. The initiative is intended to improve care largely through creation of a statewide network of regional adult SCD clinics modeled after the newly opened clinic…
LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the company’s Phase 1/2 HGB-206 clinical trial, according to the latest study data. The new findings — which included data from additional patients treated in the…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Ghana is working with Novartis to make hydroxyurea treatment available to people with sickle cell disease, and to open a clinical trial in the African country testing the company’s biologic SCD therapy Adakveo (crizanlizumab), recently approved in United States. The five-year, public-private partnership was formed in January…
Recent Posts
- Managing depression while living with sickle cell disease
- Screening tool identifies developmental risks in young children with SCD: Study
- Having my first MRI brain scan reminds me to keep advocating for myself
- Evaluating my options for treating sickle beta-zero thalassemia
- Researchers aim to repurpose diabetes drug to treat SCD kidneys
- Alzheimer’s drug memantine may help prevent SCD crises, especially in kids
- Red blood cell breakdown may drive cognitive problems in SCD
- Looking back at the 5 worst sickle cell crises of my life
- Fetal hemoglobin protects against sickle cell lung problems: Study
- Reflecting on a year of choosing myself while living with sickle cell disease