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 ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

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 ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

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 ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

Approximately 42% of patients showed evidence of SMN1 to SMN2 conversion, confirmed by the presence of SMN1-specific variants downstream of SMN2. To shed more light on the genetic determinants of SMA ...

Consolidation or maintenance therapy significantly improved progression-free survival and overall survival. Zanubrutinib, lenalidomide, temozolomide, and rituximab (RLZT) combined with or without high ...

Between 1970 and 1995, the incidence of new NHL cases increased. Between 2007 and 2016, new cases declined by approximately 0.9% per year, and mortality from NHL decreased by 2.2% per year. 3 ...

Tumor volume and CP score were top predictors of OS, along with liver gross tumor volume, functional liver volume V20, and prior liver-directed therapy. Functional liver metrics assessed using sulfur ...

The modified Spinal Muscular Atrophy Functional Rating Scale (SMAFRS) is a psychometrically stable and unidimensional outcome measure, even though certain items are misfitting like those related to ...