A diagnosis of ATTR-CM in a more recent era was associated with older age, White race, wild‐type disease, and tafamidis treatment. The survival of patients diagnosed with transthyretin-mediated ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

A pattern recognition approach takes into account factors like disease progression rate, whether the disease symptoms are symmetrical or asymmetrical, whether they are predominantly distal or proximal ...

Disease biology, not conditioning intensity, predicted outcomes following allo-HSCT for MDS with excess blasts. An age- and comorbidity-adjusted conditioning regimen achieved durable engraftment, low ...

Ofirnoflast’s NEK7 modulation offers a first-in-class anti-inflammatory approach to MDS. The US Food and Drug Administration (FDA) granted Orphan Drug Designation to ofirnoflast (HT-6184) for the ...

Recurrent pancreatitis may be a manifestation of IgG4-related autoimmune pancreatitis. A teenage boy with recurrent pancreatitis and a history of autism and learning disabilities was diagnosed with ...

Thymidine monophosphate cannot serve as a precursor for the synthesis of thymidine triphosphate (TTP) in mitochondria isolated from the liver, kidneys, and brain unless dephosphorylation to thymidine ...

Analyzing the phenotypes of patients with IgG4-related autoimmune pancreatitis can lead to more personalized treatment and, hopefully, better outcomes. Autoimmune pancreatitis (AIP) associated with ...

The updated 2025 BSR management recommendations for patients with AAV provide systematic and evidence-based recommendations to support UK clinicians in managing AAV across the entire life course.

Treatment discontinuation rates were similar to those in the placebo group, and no notable safety imbalances were observed for serious adverse events. Nerandomilast, an investigational oral ...

Extremely low baseline neutrophil count was a strong predictor of early death, underscoring the need for early risk stratification. Treatment with porcine antihuman T-lymphocyte globulin (ATG) and ...

Newborn screening remains an unevenly accessible resource. The legal and regulatory frameworks governing retention of dried blood spots used in newborn screening vary significantly across states. When ...