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 ...

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 ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

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 ...

Inherited genetic metabolic disorders, including carnitine palmitoyltransferase II (CPT-II) deficiency, should well be presumed in adults with recurrent events of rhabdomyolysis because of its swift ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

A mediation analysis revealed that 16.4% of nintedanib's effect on the change in forced vital capacity at week 52 was attributed to the treatment-induced reduction in the level of cancer antigen 125 ...

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 ...

Adults with Prader-Willi syndrome tend to age prematurely, losing muscle mass and experiencing hair graying by age 30 or 35 years. Adults with Prader-Willi syndrome (PWS) typically show gains in lower ...

The study will measure hemoglobin levels among patients taking HRS-5965 who do not require transfusions during the study period. A new Chinese study is preparing to launch that will investigate the ...

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 ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...