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Sickle Cell Disease

All articles tagged with #sickle cell disease

Long Island Reports High Sickle Cell Burden, NYU Study Finds
health28 days ago

Long Island Reports High Sickle Cell Burden, NYU Study Finds

A NYU School of Global Public Health study of 42,200 sickle cell disease hospitalizations in New York (2009–2022) finds NYC has the most admissions, but Long Island has the highest share (10%) of hospitalizations with a major risk of death and the region incurs the highest total hospital charges. The study also shows rising rates of major severity (13% to 27%) and major mortality risk (3% to 13%), with COVID-19 likely contributing to the surge. Most hospitalized patients were Black (83%), and adults 18–29 accounted for 40%. The authors call for better access to specialists, improved pediatric-to-adult transition, and policy/funding efforts to reduce regional care gaps and emergency department burden.

via New York Post|
#health#hospitalizations#long-island
CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin
health1 month ago

CRISPR-based sickle cell therapy earns $3M Breakthrough Prize for Thein and Orkin

Two researchers, Swee Lay Thein and Stuart Orkin, won the Breakthrough Prize in Life Sciences worth $3 million for work that led to Casgevy, a CRISPR-based therapy that functionally cures sickle cell disease and beta-thalassemia by disabling the BCL11A repressor to boost fetal hemoglobin. The treatment—developed with Vertex—involves editing a patient’s bone marrow cells and reinserting them, a costly, lengthy process with chemotherapy, and while a major milestone, access remains limited as scientists pursue in vivo approaches to broaden use.

via Live Science|
#bcl11a#beta-thalassemia#casgevy
Half-Matched Bone Marrow Transplant Brings Pain-Free Life to Sickle Cell Patient
health2 months ago

Half-Matched Bone Marrow Transplant Brings Pain-Free Life to Sickle Cell Patient

A woman diagnosed with sickle cell disease at 2 months old is cured after a haploidentical bone marrow transplant from her 50% genetic match at Johns Hopkins; she woke up pain-free the same day and by January 2025 was off pain medications, with doctors citing a 94% disease-free survival rate for this approach, signaling a potential cure for many sickle cell patients.

via Yahoo News Canada|
#disease-free-survival#haploidentical-bone-marrow-transplant#health
CRISPR breakthrough activates genes without DNA cuts
science4 months ago

CRISPR breakthrough activates genes without DNA cuts

Scientists at UNSW Sydney have developed a new CRISPR-based epigenetic editing technique that can turn genes on without cutting DNA, by removing chemical methyl groups that silence genes. This approach could lead to safer gene therapies for conditions like Sickle Cell disease, as it avoids the risks associated with DNA strand breaks. The research demonstrates that methylation directly controls gene activity and opens new possibilities for treating genetic disorders by reactivating silenced genes.

via ScienceDaily|
#crispr#dna-methylation#epigenetic-editing
Community Efforts and Advances in Sickle Cell Disease Awareness and Support
health8 months ago

Community Efforts and Advances in Sickle Cell Disease Awareness and Support

The article tells the story of Aiden-Jon Amir Hall, a young boy with sickle cell anaemia who passed away at age five, and his mother Latoya Minott Smikle's efforts to raise awareness and funds through the Aiden-Jon Amir Hall Foundation to support others affected by the disease, highlighting the challenges faced by families and the need for greater resources and support.

via Jamaica Observer|
#aiden-jon-amir-hall#awareness#foundation
Debate Over US Foreign Aid Cuts and Their Humanitarian Impact
world1 year ago

Debate Over US Foreign Aid Cuts and Their Humanitarian Impact

Mariam Mohammed blames U.S. aid cuts for her son Babagana's death in Nigeria, highlighting the human toll of reduced foreign assistance, despite claims by officials like Marco Rubio that no lives have been lost due to these cuts. The story underscores the impact on healthcare services in vulnerable regions and estimates suggest thousands have died as a result of aid reductions.

via NPR|
#babagana-mohammed#healthcare#nigeria
Editas Medicine Restructures, Cuts 65% of Workforce to Focus on In Vivo Gene Editing
business1 year ago

Editas Medicine Restructures, Cuts 65% of Workforce to Focus on In Vivo Gene Editing

Editas Medicine announced it will lay off 65% of its workforce, approximately 180 employees, as it discontinues its lead gene-editing program for sickle cell disease. This decision comes amid a challenging financial period for the gene-editing industry, with declining valuations and widespread layoffs. Despite its promising start as a CRISPR pioneer, Editas has faced execution challenges and significant turnover, leading to this strategic shift.

via STAT|
#business#crispr#editas-medicine
Sanofi's Rilzabrutinib Shows Promise in Treating Rare Platelet Disorder
health1 year ago

Sanofi's Rilzabrutinib Shows Promise in Treating Rare Platelet Disorder

Sanofi's rilzabrutinib, an oral treatment for immune thrombocytopenia (ITP), showed promising results in a Phase 3 trial, improving platelet response and reducing bleeding episodes. The drug, a bruton's kinase inhibitor, is safer and better tolerated than existing treatments, potentially setting a new standard for ITP therapy. Meanwhile, Novo Nordisk's etavopivat reduced severe pain crises in sickle cell disease patients in a Phase 2 study, though results were not statistically significant. Beam Therapeutics' CRISPR-based therapy for sickle cell disease also showed consistent positive results, with no severe pain crises reported among treated patients.

via STAT|
#health#immune-thrombocytopenia#novo-nordisk
Navigating Sickle Cell Challenges and Resilience in America
health1 year ago

Navigating Sickle Cell Challenges and Resilience in America

Tammy Clemons, a 41-year-old woman with sickle cell disease, navigates the challenges of her condition while dealing with personal loss and family crises. Despite frequent hospital visits and the stigma surrounding her illness, she finds solace in her work at a beauty supply store and support from her doctor, Dr. Sana Saif Ur Rehman, who specializes in sickle cell treatment. Clemons' story highlights the complexities of living with a chronic illness and the importance of compassionate healthcare.

via STAT|
#family#health#healthcare