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New study: high efficiency of severe thalassemia prevention with HTS based carrier screening

A recent study found that strict adherence to High-Throughput Sequencing (HTS) technology based carrier screening can achieve high efficiency in preventing severe thalassemia birth defects. The study identified 15.07% of women as carriers of thalassemia and confirmed 59 fetuses with severe thalassemia, all of which were in high-risk co...

SourceBGI Genomics·JournalJournal of Genetics and Genomics·DateMay 8, 2025

A new CAR-T therapy from Sant Pau achieves positive results in a high proportion of patients with a refractory type of lymphoma

A new CAR-T therapy, HSP-CAR30, has achieved positive results in a high proportion of patients with refractory CD30+ lymphoma. The treatment promotes the expansion of memory T cells, leading to durable responses and improved clinical outcomes.

SourceInstitut de Recerca Sant Pau (Sant Pau Research Institute)·JournalBlood·TypeRandomized controlled/clinical trial·DateApr 29, 2025

Minimal TV viewing may be protective for heart diseases linked to Type 2 diabetes

A new study found that limiting TV viewing to one hour or less daily may help offset an increased risk of heart and blood vessel diseases for people with a genetic risk for Type 2 diabetes. This suggests that reducing TV watching could serve as a key behavioral target for preventing atherosclerotic cardiovascular diseases linked to Typ...

SourceAmerican Heart Association·JournalJournal of the American Heart Association·DateMar 12, 2025

Cutting back on alcohol may be more effective than immediately abstaining for preventing misuse among college students

A Texas A&M University researcher found that reducing alcohol consumption aligns with a harm reduction approach, which may be a more feasible approach for high-risk college drinkers. The study surveyed 822 high-risk college students and analyzed their intention to reduce drinking using the Theory of Planned Behavior.

SourceTexas A&M University·JournalAmerican Journal of Health Education·DateJan 30, 2025

Houston Methodist scientists make surprising discovery pinpointing when good cholesterol becomes harmful

A study led by Henry J. Pownall and Khurram Nasir found a strong link between the amount of free cholesterol in HDL and its accumulation in macrophages, which can contribute to heart disease. The researchers aim to develop new diagnostics and treatments for managing heart disease using HDL-free cholesterol as a biomarker.

SourceHouston Methodist·JournalJournal of Lipid Research·TypeExperimental study·DateDec 19, 2024

Children’s Hospital Colorado research transforms the standard of care for childhood cancer

The new therapy significantly improves three-year disease-free survival for children with B-ALL, with 96% being disease-free for at least three years. Blinatumomab is now being used in most patients due to its practice-changing breakthrough, offering improved quality of life and reduced side effects.

SourceChildren's Hospital Colorado·JournalNew England Journal of Medicine·TypeRandomized controlled/clinical trial·DateDec 18, 2024

ASH 2024: Antibody shows encouraging results for treating high-risk follicular lymphoma and marginal zone lymphoma

Two clinical trials testing loncastuximab tesirine showed promising results in patients with high-risk forms of follicular lymphoma and marginal zone lymphoma. The treatment achieved impressive response rates, including a 70% complete response rate for marginal zone lymphoma and an overall response rate of 97% for follicular lymphoma.

SourceUniversity of Miami Miller School of Medicine·JournalThe Lancet Haematology·DateDec 8, 2024

Intrinsic factors of the CAR-T cells may play a major role on its clinical success

A new study found that CAR-T cell therapy success is predicted by three key features: the CD4/CD8 ratio in infusion products, the presence of T-cell exhaustion signals before infusion, and the expansion of T-cells during therapy. These factors can help improve treatment outcomes.

SourceJosep Carreras Leukaemia Research Institute·JournalCell Reports Medicine·TypeComputational simulation/modeling·DateOct 28, 2024

Kumamoto University scientists achieve successful reproduction of hematopoietic stem cell developmental process in an in vitro culture system

Researchers at Kumamoto University have achieved a groundbreaking advancement in stem cell biology by reproducing the developmental process of hematopoietic stem cells in vitro. This culture system enhances our understanding of HSC development and has the potential to be instrumental in stem cell therapy and blood disease treatments.

SourceKumamoto University·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateOct 18, 2024

Scientists discover potential cause of an enigmatic vascular disease primarily impacting women

Researchers identified changes in the UBR4 gene as a key driver of fibromuscular dysplasia, a blood vessel disorder affecting up to five percent of adults. The discovery could lead to therapeutic approaches for the condition, particularly beneficial for women who account for 90% of cases.

SourceThe Mount Sinai Hospital / Mount Sinai School of Medicine·JournalNature Cardiovascular Research·TypeExperimental study·DateSep 13, 2024

NUS artificial intelligence platform demonstrates promising results in effectively treating a patient with a rare cancer

A team at NUS Yong Loo Lin School of Medicine leveraged an artificial intelligence-derived platform, CURATE.AI, to guide treatment for a patient with Waldenström macroglobulinemia, a rare blood disorder. The trial demonstrated substantial improvement in red blood cell levels and minimised side effects.

How a protein component of nuclear pore complexes regulates development of blood cells and may contribute to myeloid disorders

A recent study has identified Nup358 as a critical regulator of myeloid cell development, revealing its role in the differentiation process of early progenitors. The findings provide insights into how alterations in Nup358 contribute to blood malignancies and may lead to novel therapies targeting transport machinery like NPCs.

SourceSanford Burnham Prebys·JournalScience Advances·TypeExperimental study·DateJun 5, 2024

Gentler cell therapies for blood cancer

Researchers have developed an approach to 'delete' a diseased blood system while building up a new, healthy one with donor blood stem cells. This process involves targeting specific antibodies coupled to a cytotoxic drug that recognize and destroy diseased blood cells while sparing healthy ones.

SourceUniversity of Basel·JournalNature·TypeExperimental study·DateMay 22, 2024

Chinese Medical Journal review article highlights the potential and promise of CAR-T cell therapy in autoimmune diseases

Researchers have identified CAR-T cell therapy as a potential treatment for autoimmune diseases such as rheumatoid arthritis, SLE, and type 1 Diabetes Mellitus. Early studies have shown promising results in reducing disease activity and improving patients' quality of life, but long-term data on safety and efficacy is limited.

SourceCactus Communications·JournalChinese Medical Journal·TypeLiterature review·DateApr 22, 2024

UMSOM researchers identify safety of a potential new treatment to manage complications from sickle cell disease

Researchers found that riociguat significantly improved blood pressure and reduced the risk of serious adverse events compared to a placebo treatment. The study showed promising results for this potential new treatment to manage complications from sickle cell disease, paving the way for larger clinical trials.

SourceUniversity of Maryland School of Medicine·JournalThe Lancet Haematology·TypeRandomized controlled/clinical trial·DateApr 11, 2024

Two new mutations identified as possible causes of rare blood disorder

Researchers discovered two novel GNE gene mutations that may cause a rare blood disorder called macrothrombocytopenia. The mutations affect the synthesis of sialic acid, critical for brain development and angiogenesis. Further studies are needed to understand the mechanism underlying this disorder and explore therapeutic interventions.

Children’s Hospital Los Angeles study finds many kids with sickle cell anemia lack preventative care

A study by Children's Hospital Los Angeles found that only about 20% of young children with sickle cell anemia received adequate preventative antibiotics, while about half received annual brain ultrasounds to assess stroke risk. These findings highlight the need for improved care and support for children with this chronic disease.

SourceChildren's Hospital Los Angeles·JournalPEDIATRICS·TypeData/statistical analysis·DateMar 6, 2024

Latest research redefines neurodevelopmental risks, outcomes for congenital heart disease

A new American Heart Association scientific statement offers the latest knowledge on neurodevelopmental outcomes in people of all ages with congenital heart disease. The statement highlights that neurodevelopmental difficulties are common complications faced by individuals with congenital heart disease, affecting their ability to funct...

SourceAmerican Heart Association·JournalCirculation·DateFeb 22, 2024

Deaths from heart valve infections drop across U.S. overall, but surged among young adults

A new study found that deaths from infective endocarditis decreased overall in the US, but rose sharply among people ages 25-44, likely linked to the opioid crisis. Researchers call for more investigation into the trends and recommend comprehensive care plans including substance use disorder screening and treatment.

SourceAmerican Heart Association·JournalJournal of the American Heart Association·DateDec 13, 2023

'Exceptional' results in phase III leukaemia trial

A phase III trial has found that personalised treatment for chronic lymphocytic leukaemia (CLL) can improve survival and remission rates. The trial showed that individualising therapy based on regular blood tests significantly improved progression-free and overall survival in patients with previously untreated CLL.

SourceUniversity of Leeds·JournalNew England Journal of Medicine·DateDec 10, 2023

Long-term results show combination treatment that skips chemotherapy is effective for older patients with Ph+ ALL

The study found that dasatinib and blinatumomab combination treatment resulted in excellent median overall survival and disease-free survival times for patients 65 and older with Philadelphia chromosome-positive acute lymphoblastic leukemia. The regimen confirmed benefits of this treatment are durable, with a median overall survival of...

SourceSWOG Cancer Research Network·TypeRandomized controlled/clinical trial·DateDec 9, 2023

Small but mighty: Microparticles from the placenta may offer major clues on the in utero development of neurobehavioral disorders

Researchers discovered how biological information travels between mothers and babies during pregnancy. Microscopic extracellular vesicles, produced by placental cells, act as a protective mechanism transporting important biological information to emerging neurons.

SourceUniversity of Missouri-Columbia·JournalBiology of Reproduction·TypeExperimental study·DateDec 5, 2023

Leukemia cells activate cellular recycling program

A recent study by Goethe University Frankfurt has identified a mechanism that could be a suitable starting point for developing novel drugs against leukemia cells. The researchers discovered that the mutated NPM1 gene variant drives pro-autophagic activity, enabling cancer cells to recycle their structures and meet their needs.

SourceGoethe University Frankfurt·JournalCell Reports·TypeExperimental study·DateDec 4, 2023

Montefiore Einstein Comprehensive Cancer Center awarded FDA grant for clinical trial on experimental AML/MDS treatment

Researchers at Montefiore Einstein Comprehensive Cancer Center are conducting a phase 1 clinical trial of danvatirsen, a STAT3 inhibitor, to treat relapsed and treatment-resistant forms of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The trial aims to determine the safety and efficacy of the experimental treatment ...