Sickle Cell Disease News Home | Sickle Cell Disease News
Oct 16, 2024 · Editas’ new gene-editing strategy for SCD shows promise in mice. Editas Medicine has announced proof-of-concept data for a new gene-editing approach for sickle cell disease (SCD) that does not involve a stem cell transplant. The strategy instead makes use of targeted lipid nanoparticles, or LNPs, which are tiny vesicles made of fatty molecules.
Vaso-Occlusive Crisis - Sickle Cell Disease News
Jan 4, 2022 · A vaso-occlusive crisis, or VOC, occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen. This in turn sets in motion an inflammatory response as the body tries to rectify the problem. The result is substantial pain, which can affect any part of the body, but most commonly occurs in the back ...
Oral AND017 granted orphan drug status for SCD
5 days ago · by Margarida Maia, PhD October 31, 2024. The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S.
Phase 3 RISE UP study of mitapivat for SCD completes enrollment
Oct 24, 2024 · Results from 79 patients who took part in the Phase 2 dose-finding study, announced late last year, indicated the two tested doses of mitapivat (50 or 100 mg taken twice daily) could boost hemoglobin levels compared with a placebo over the course of about three months.Both doses also led to reductions in the annualized rate of pain crises. In the Phase 3 study, participants will be randomly ...
Gene Therapy – Sickle Cell Disease News
Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene.These Phase 2 trials are recruiting both children and adults in the United States and Jamaica. Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use of Mozobil (plerixafor) in patients with sickle ...
How I manage mild to moderate sickle cell pain at home
6 days ago · First dose of ibuprofen at 11 a.m. Second dose of acetaminophen at 2 p.m. Second dose of ibuprofen at 5 p.m. Third dose of acetaminophen at 8 p.m. Oral opioids like dihydrocodeine, morphine, and tramadol are reserved for moderate to severe pain and should be used only with a doctor’s prescription. Heat therapy and topical analgesics also work ...
FDA clears study of potentially less toxic SCD conditioning regimen
Jul 30, 2024 · The U.S. Food and Drug Administration (FDA) gave the greenlight for Iomab-ACT, a potentially less toxic conditioning regimen, to be tested in people with sickle cell disease (SCD) undergoing a stem cell transplant. The study will be conducted in collaboration between Actinium Pharmaceuticals, which is developing Iomab-ACT, and Columbia ...
News Archives - Sickle Cell Disease News
The number of stroke cases among adults and children with sickle cell disease (SCD) continues to increase in at least one U.S. state, indicating a need for greater adherence to standard-of-care blood transfusions and more attention paid to factors that can be controlled to reduce the risk of…. October 1, 2024 News by Steve Bryson, PhD.
Risk of blood clots more likely with sickle cell trait, but still low
Sep 19, 2024 · Still, the researchers said, the risk of these blood clots was low, occurring in just over 2% of people carrying SCT. The study, “ Ancestry-Independent Risk of Venous Thromboembolism in Individuals with Sickle Cell Trait vs. Factor V Leiden,” was published in Blood Advances. “I hope this study informs health professionals, particularly hematologists, that when thinking about risks ...
Sickle Cell 101: Addressing Common Questions About My Blood …
Sep 10, 2019 · Sickle cell is an inherited disorder that affects the blood’s ability to carry oxygen. The red blood cells in my body are shaped like a “C” instead of the typical “O.”. A consequence of having these sickle-shaped red blood cells is that they can stick together, which restricts the flow in my blood vessels. This causes severe pain ...