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 ...

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 ...

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, ...

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 ...

As of November 2024, approximately 137 clinical trials for spinal muscular atrophy (SMA) are ongoing or pending: 8 trials that are not yet recruiting, 74 trials that are currently recruiting, 26 ...

Just over half of intrahepatic cholangiocarcinoma (iCCA) specimens in a North American cohort could be classified into small duct and large duct subtypes using morphology alone, according to study ...

Thymidine kinase 2 deficiency (TK2d) is a very rare inherited mitochondrial disease characterized by a gradual reduction of the amount of genetic material within mitochondria. Patients with TK2d, ...

The first case of sickle cell disease (SCD) in the United States was described in 1904 when Walter Clement Noel, a 20-year-old dental student from Grenada, sought care for anemia at Chicago ...

When comparing the digital and paper formats of the eMPhasis-10, the responses from patients showed remarkable consistency. In other words, there was a strong association between both digital and ...

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.

As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending: 16 trials not yet recruiting, 65 currently recruiting, 28 trials that are active ...