Selinexor in combination with ruxolitinib achieved a statistically significant improvement in spleen volume reduction for patients with MF, according to a press release. Rare Disease Advisor, a ...

In a cross-sectional study, the researchers reported psychiatric comorbidities in 46.5% of 43,270 adults with neurofibromatosis. The most common psychiatric comorbidities included mood disorders (22.1 ...

The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...

Dentists should be aware that manifestations of hereditary angioedema may be triggered by certain dental procedures. A boy who presented for dental treatment experienced periorbital and facial ...

Castleman disease and IgG4-RD have overlapping disease manifestations that can complicate the diagnostic process. A male patient initially suspected of having immunoglobulin G4-related disease ...

When I went out a few days ago, I noticed everyone had a runny nose or a cough. The weather here in Ohio has been chilly, about 30 °F (-1 °C), but we haven’t had our usual January extreme cold yet.

Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by the pathological activation of the terminal complement pathway, which causes intravascular hemolysis and thrombosis. Untreated, it is ...

Amy Comstock Rick of the US Food and Drug Administration speaks at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference (Photo by Riya Ajmera) Due to differences in the nature of ...

Cardiomyopathy with systolic heart failure is the leading cause of death in Duchenne muscular dystrophy (DMD), confirmed a new longitudinal observational study published in ERJ Open Research. Moreover ...

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 ...

I was supposed to be dead by now, or so statistics said. In 1980, when I was diagnosed with multiple sclerosis (MS), the life expectancy of an American man was a little over 73 years. After my ...