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Nov 1, 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.
More stem cells for sickle cell gene therapy mobilized with...
1 day ago · It may be possible to collect nearly three times as many stem cells for gene therapy for sickle cell disease (SCD) with BioLineRx ’s motixafortide than with standard plerixafor, and even more when it’s combined with natalizumab, a Phase 1 study shows. The proof-of-concept clinical trial (NCT05618301) is testing how safe and well tolerated ...
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 ...
News Archives - Sickle Cell Disease News
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.
Study to compare SCD treatments for symptoms vs. a transplant
4 days ago · Particularly, the study by scientists with Golisano Children’s Hospital will compare sickle cell treatments that help to control disease symptoms, such as blood transfusions and hydroxyurea, with those aiming for a cure, like bone marrow transplantation. The project aims to recruit nearly 500 children and young adults with SCD, who either ...
Oral AND017 granted orphan drug status for SCD
Oct 31, 2024 · by Margarida Maia, PhD October 31, 2024. The U.S. Food and Drug Administration 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.
New Assistance Fund program will help with SCD medical costs
May 23, 2023 · The Assistance Fund (TAF) has launched a new financial assistance program to cover out-of-pocket medical expenses for people with sickle cell disease (SCD). The program will cover copayments, health insurance premiums, costs related to ground travel needed to get treatment, and genetic testing, for people with SCD who are underinsured and face ...
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 ...
AB Science gets European patent covering masitinib for SCD
Nov 5, 2024 · AB Science said it received a notice of allowance from the European Patent Office for a patent covering the use of the experimental oral therapy masitinib to treat sickle cell disease (SCD). The decision was based on preclinical data demonstrating that masitinib was able to prevent vaso-occlusive crises (VOCs), a common complication of the ...
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About Sickle Cell Disease News. Sickle Cell Disease News is a digital platform intended to provide the sickle cell disease (SCD) community with the most recent news and information on SCD, as well as first-hand community perspectives from our patient and caregiver columnists. All articles on Sickle Cell Disease News are original content ...