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Sickle Cell Disease

All articles tagged with #sickle cell disease

science9 days 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
health3 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
world7 months 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
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
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
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
health1 year ago

Vertex Sues US for Fertility Support in Casgevy Gene Therapy

Vertex Pharmaceuticals has filed a lawsuit against the U.S. government to allow the company to fund fertility preservation services for patients receiving Casgevy, a CRISPR-based treatment for sickle cell disease. The lawsuit challenges an oral opinion from the Health and Human Services Office of the Inspector General, which deemed the program a violation of federal anti-kickback laws. Vertex argues that the program is essential for patients who must undergo chemotherapy, which can cause infertility, and that the refusal to allow such support exacerbates healthcare inequalities.

via STAT|
#anti-kickback-laws#crispr#fertility-preservation
health1 year ago

Sickle Cell Patients Face Reproductive Health Coercion Amid Abortion Bans

A woman with sickle cell disease was denied adequate pain medication during a crisis at a Texas hospital because she was pregnant, and a doctor offered her pain relief only if she agreed not to get an abortion. Facing severe pain and fearing for her life, she eventually traveled to Boston for an abortion. This case highlights the conflicting pressures and ethical dilemmas faced by patients with sickle cell disease, especially in states with strict abortion laws.

via STAT|
#abortion#health#medical-ethics
health1 year ago

"New Gene Therapy Brings Hope for Sickle Cell Cure in the US"

A 12-year-old boy named Kendric Cromer has become the first patient in the U.S. to undergo a newly approved gene therapy for sickle cell disease at Children's National Hospital in D.C. The treatment, which involves modifying his bone marrow stem cells, offers hope for a potential cure and could transform care for thousands suffering from the debilitating condition. Kendric and his family are optimistic about the future, despite the painful and costly process.

via NBC Washington|
#childrens-national-hospital#gene-therapy#health
healthcare-gene-therapy-sickle-cell-disease-medicaid-healthcare-access1 year ago

"Addressing the Cost Barrier for Breakthrough Sickle Cell Gene Therapies"

The approval of two gene therapies to treat sickle cell disease has brought hope to patients, but the high cost of the treatments poses a challenge for equitable access. With prices ranging from $2.2 million to $3.1 million, patients like Michael Goodwin are hesitant due to the intensive medical preparation and concerns about affordability. State and federal officials are negotiating with drugmakers to obtain discounts for Medicaid plans, while employers are exploring new payment models to cover the rising costs of specialty treatments. The lack of infrastructure and the high cost of the treatments raise questions about how many people will benefit from them.

via CNBC|
#gene-therapy#healthcare-access#healthcare-gene-therapy-sickle-cell-disease-medicaid-healthcare-access