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Nov 1, 2024 · BEAM-101 gene-editing stem cell therapy shows promise in SCD. In a Phase 1/2 clinical trial, the gene-editing stem cell therapy BEAM-101 worked as intended, and showed early signs of efficacy in people with severe sickle cell disease (SCD), according to new early data.
Vaso-Occlusive Crisis - Sickle Cell Disease News
Jan 4, 2022 · Sickle cell disease (SCD) is an inherited disorder caused by a mutation in the gene encoding a subunit of hemoglobin, the protein that carries oxygen in red blood cells. Mutant hemoglobin — called sickle hemoglobin or hemoglobin S — tends to clump together in red blood cells, causing them to take on an irregular “sickle-like” shape that gives the disease its name.
Oxbryta's withdrawal is a setback in the fight against sickle cell
Oct 14, 2024 · Pfizer’s recent decision to withdraw Oxbryta (voxelotor) from global markets feels like a devastating blow for many people with sickle cell disease. Oxbryta’s conditional approval in 2019 by the U.S. Food and Drug Administration gave hope to many, as few treatments exist for patients with sickle ...
Study to compare SCD treatments for symptoms vs. a transplant
5 days ago · A bone marrow transplant, in which hematopoietic stem cells responsible for giving rise to new blood cells are replaced by those derived from a healthy donor, is an invasive treatment but one also seen as a possible cure of SCD.However, the donor must match the patient’s human leukocyte antigens, a set of proteins …
Here's what we can do for National Sickle Cell Awareness Month
Sep 16, 2024 · These activities help to build a sense of community among people with sickle cell and to educate the broader public. The stories that are shared help to put a human face on the disease, moving it from simply a clinical diagnosis to a broader understanding of the daily struggles and triumphs of those who live with it.. With all of this in mind, it’s also a time to celebrate the resilience and ...
Stroke rate rising for adults, children with SCD in US: Study
Oct 3, 2024 · Guidelines from 1998 STOP study intially led to lower stroke rate. People with SCD have abnormally shaped red blood cells and often experience strokes — which happen when blood flow to the brain is blocked or a blood vessel bursts — and transient ischemic attacks, where the blockage is temporary and doesn’t cause lasting damage.
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 ...
Taking a holistic approach to sickle cell disease management
Jul 3, 2023 · Dunstan Nicol-Wilson Dunstan Nicol-Wilson is a clinical project manager from South East London, United Kingdom. Dunstan has his master’s in public health with a global health focus. Dunstan was diagnosed with the “invisible disorder” sickle cell disease from birth in 1993.
Low-intensity stem cell transplants may benefit SCD patients: Study
Sep 3, 2024 · “By using a low-intensity blood stem cell transplant for sickle cell disease, we may be able to stop the cycle of lung injury [seen in SCD patients] and prevent continued damage,” Parker Ruhl, MD, an associate research physician and pulmonologist at the NIH, and the study’s first author, said in an agency press release.. These findings may prove informative for people with SCD who may be ...
How Diet Can Help Support Sickle Cell Patients: A Nutritionist’s View
Oct 3, 2019 · A nutritional intervention to consider is a high protein diet (about 35% total energy intake) that can lessen the likelihood of infection. We have seen this specifically with diets rich in the amino acid arginine.. Argenine’s benefits for SCA patients come via its metabolite nitric oxide, which works to reduce pain and vaso-occlusive crisis (VOC) and increase muscle function, according to a ...