Sickle cell disease (SCD) is an inherited blood disorder marked by defective hemoglobin. It causes red blood cells (RBCs) to deform into a sickle shape, which results in premature death of the RBCs, ...

Follow Rare Disease Advisor's Sickle Cell Disease Junction for the latest news, resources, insights, and patient perspectives on the disease.

The term muscular dystrophy encompasses a group of genetic diseases characterized by progressive muscle weakness and wasting. There are different types of muscular dystrophy depending on genetic ...

Clinicians who treat cystic fibrosis can stay up to date on the latest news and perspections about the disease here on RDA's CF Junction.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening disorder characterized by the premature breakdown of erythrocytes and the release of hemoglobin into urine for excretion.

Diffuse large B-cell lymphoma (DLBCL) is a type of cancer that originates in and affects the lymphatic system. 1 It is the most common type of non-Hodgkin lymphoma (NHL), accounting for approximately ...

Modern prophylactic therapies for hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) have become increasingly prevalent in Hungary, paralleling global trends, and are proving both ...

Weight loss may be intensified by treatment-related side effects of diffuse large B-cell lymphoma (DLBCL), such as loss of appetite, diarrhea, 1 vomiting, 1,2 nausea, and changes in taste and smell. 2 ...

A case of acute lymphoblastic leukemia (ALL) complicated by bullous pemphigoid (BP) presented significant diagnostic and therapeutic challenges, emphasizing the need for prompt identification and ...

Researchers from Brazil developed a new clinical score to estimate the probability of neuromyelitis optica spectrum disorder (NMOSD) in patients presenting with optic neuritis. The easily accessible ...

Pulmonary arterial hypertension (PAH) is a severe, chronic pulmonary arteriolar disease characterized by increased pulmonary vascular resistance. It affects functional capacity and quality of life, ...

Clinically relevant concentrations of deoxycytidine (dC) and deoxythymidine (dT) are unlikely to cause or be subject to pharmacokinetic drug-drug interactions, according to a new study published in ...