In patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), high levels of baseline blood urea nitrogen (BUN) are a risk factor for end-stage kidney disease (ESKD) and/or ...

The presence of intracutaneous amyloid deposition in asymptomatic TTR mutation carriers could be a predictive of ATTR-CM progression.

Age over 75 years was significantly associated with reduced survival and shorter time on treatment. Ibrutinib remains an effective therapy for chronic lymphocytic leukemia (CLL), but older patients ...

The change in extracellular volume is the only predictor of adverse outcome in patients with ATTR-CM treated with tafamidis, a study found.

Özge’s background is in research; she holds a MSc. in Molecular Genetics from the University of Leicester and a PhD. in Developmental Biology from the University of London. Özge worked as a bench ...

Therapies for Gaucher disease generally fall into 2 main categories: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Neither ERT nor SRT effectively treats the neurological ...

Isogenic induced pluripotent stem cell models carrying Gaucher disease-linked GBA1 variants exhibited reduced GCase RNA, protein, and enzymatic activity.

Immune-mediated hypertrophic meningitis (HP) is associated with diverse etiologies, with the main causes being immunoglobulin G4-related disease (IgG4-RD) HP, antineutrophil cytoplasmic antibody (ANCA ...

Are you a health care provider looking for information on experimental therapies for hepatocellular carcinoma (HCC)? Read more here.

A recently established committee will discuss a novel therapeutic option for Duchenne muscular dystrophy (DMD), according to a press release made by Sarepta Therapeutics, Inc. “It is our understanding ...

Frequent non-invasive hemoglobin monitoring via a smartphone app can effectively predict vaso-occlusive crises in sickle cell disease (SCD).

Angela Vincent, MBBS, a British neurologist and "fixture at the front of myasthenia gravis research for 50 years," is the award's first recipient.