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 ...

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

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, ...

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 ...

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 risk of pregnancy complications and adverse birth outcomes has been observed in mothers with alpha-1 antitrypsin deficiency (AATD), according to a study recently published in Plos One. The ...

Sheila Jacobs is the president of STJ Editorial Services. For more than 25 years, Sheila has been providing writing, fact-checking, and editorial services to numerous clients in the medical education, ...

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.

Patients with alpha-1 antitrypsin deficiency (AATD) receiving inhaled ATT therapy have demonstrated significant improvements in restoring protease–antiprotease homeostasis, according to results of a ...

A patient with chronic lymphocytic leukemia (CLL) achieved complete response after beginning methionine restriction (MR) in combination with chemotherapy. This case report was published in Cancer ...

The CSHA Clinical Frailty Scale (CFS) is a valuable tool for predicting treatment-related toxicity and discontinuation in older adults receiving targeted therapy for chronic lymphocytic leukemia (CLL) ...

Which of the statements below about the complications of immunoglobulin G4-related disease (IgG4-RD) are correct? Select one of the options for each question, then submit your answers to see how well ...