Intellia Therapeutics has used gene editing to replace an oxygen-carrying component in blood that is defective in sickle cell disease. The work involved editing the BCL11A gene in a mouse model of the disease to produce an effective version of the oxygen-carrying component, hemoglobin. Intellia discussed the project at the…
News
Sickle cell disease patients who have relatively low pain levels use opioids more when they are sad, anxious, or experience other negative emotions, a study indicates. The Johns Hopkins University research was based on entries in 45 patients’ electronic diaries. It suggested that patients misused opioids when they increased their intake…
A new online platform, called oneSCDvoice.com, will help those affected by sickle cell disease (SCD) by offering free access to comprehensive support and a robust knowledge network . rareLife solutions, the health tech, design and research team behind oneSCDvoice and other custom rare disease platforms, will launch the modular community…
Anyah Randolph, a teenager with sickle cell disease (SCD), was recently able to join her school class on a trip to Europe – and even climb the Eiffel Tower in Paris after the elevator broke – thanks to the help of hydroxyurea and Loyola Medicine pediatricians. Anyah had…
Following a review of studies published in the last decade, experts have updated their recommendations for managing a sickle cell disease-related eye complication known as sickle cell retinopathy (SCR). Their paper, “Sickle cell retinopathy: improving care with a multidisciplinary approach,” appeared in the Journal of Multidisciplinary Healthcare. SCR, a…
An independent review board has found Global Blood Therapeutics’ sickle cell disease treatment voxelotor safe, and recommended that its Phase 3 clinical trial continue. The hallmark of sickle cell disease is damage to a red blood cell protein called hemoglobin that carries oxygen to organs and tissue. The damage causes the cells…
The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…
Howard University researchers have discovered a link between increases in Veillonella gut bacteria and higher levels of pain in sickle cell disease patients. The team presented their findings at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, Nov. 6-8. Sickle cell disease is a blood…
Global Blood Therapeutics (GBT) reported a case study in which a sickle cell disease (SCD) patient was granted compassionate access to the company’s ongoing Phase 3 HOPE study of voxelotor (NCT03036813). The patient had severe, transfusion-refractory anemia and experienced rapid improvements with the therapy.
Memantine, a standard treatment for Alzheimer’s disease, has shown promise in treating sickle cell disease (SCD) in six patients, a 12-month pilot clinical trial found. The findings were presented at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, D.C. The study’s lead researcher,…
Recent Posts
- 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
- New year, old goals, because repeating health resolutions works for sickle cell
- Researchers compare gene therapies for sickle cell disease in mice
- Emmaus to sell North American rights to sickle cell treatment Endari
- For sickle cell patients, developing self-advocacy skills starts in childhood
- Standard sickle cell treatments slash stroke risk in children: Review