New Rochelle, NY – A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies against either adeno-associated virus (AAV) 2 or AAV9. Click here to read the article now. NPC1 is a rare, fatal neurodegenerative...
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ATLANTA — New research at ACR Convergence 2023, the American College of Rheumatology’s (ACR) annual meeting, found that rheumatoid arthritis (RA) patients in sustained remission who stopped TNF inhibitors (TNFi) had significantly more flares and lower Boolean 2.0 remission rates compared with those who continued treatment. Boolean 2.0 is a...
ANN ARBOR, Mich. — At the ends of chromosome are special pieces of DNA called telomeres. Think of it as the little tip that caps off a shoelace. The telomeres send signals to the cells to let them know it’s the end point, not a break that should be repaired....
Wellington, New Zealand – Researchers at the University of Otago’s Department of Paediatrics and Child Health think that the analysis of intronic regions through various methods could uncover novel variants responsible for causing Dravet syndrome (DS), according to a study recently published in Epilepsia Open. “We demonstrate that targeted interrogation of...
Boston, Mass. – A new study, “Timing of Orthostatic Hypotension and its Relationship with Falls in Older Adults”, has brought critical insights into the assessment of orthostatic hypotension, a drop in blood pressure commonly found among older adults when transitioning from a sitting or lying position to a standing position...
MIAMI, FLORIDA – Many people with stage II or III colon cancer receive additional, or adjuvant, chemotherapy following surgery. However, clinical trials have shown that this treatment doesn’t improve the chances of survival for every patient. A study published July 25 in Cell Reports Medicine identifies and validates a 10-gene...
Among some pediatric patients with acute lymphoblastic leukemia (ALL), several factors, including prephase dexamethasone and using total intravenous anesthesia during intrathecal therapy, are linked with improved central nervous system (CNS) control, according to research published in Blood. Overall survival (OS) rates have greatly improved among pediatric patients with ALL over the...
Fuzhou, China – Blocking a signaling pathway in certain white blood cells may prevent blood clots from obstructing blood flow in the pulmonary arteries — the cause of chronic thromboembolic pulmonary hypertension (CTEPH). That’s according to a new study that investigated whether a certain immune signaling pathway in the body plays...
Boston, Mass. – Programmed cell death 1 (PD-1) is an important target for immune checkpoint inhibitor therapies that block its signaling and boost T-cell activity. PD-1 inhibitors have been approved for treating various types of cancer. But PD-1 functions can vary between different cell and cancer types, either promoting or...
Zürich, Switzerland – Sjögren’s disease is rare in children, and presenting symptoms differ from those seen in adults. For example, paediatric patients present less often with sicca complaints, and more frequently with parotid gland swelling and fever.1 This new work aimed to identify potentially dysregulated molecular pathways in children with...