Assessing Epidemiological Data Points The findings from this study echo that of another by Trivella and colleagues, also published in Hepatology Communications. This 2023 study is a literature review ...

There are several experimental therapies under investigation for the treatment of Fabry disease, including enzyme replacement therapy (ERT), substrate reduction therapy (SRT), and gene therapy. 1 ...

Amyloid transthyretin cardiomyopathy (ATTR-CM) is a progressive and life-threatening heart disease that can lead to increased ventricular wall thickness, restrictive cardiomyopathy, and heart failure.

Two phase 3 clinical trials, MOBILIZE and VITALIZE, are recruiting patients with chronic demyelinating polyneuropathy (CIDP) to evaluate the effectiveness of riliprubart according to an abstract ...

A single intravenous infusion of the investigational adeno-associated virus (AAV) gene therapy scAAV2/8-LP1-hFIXco demonstrated durable clinical benefit and long-term safety in patients with severe ...

Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult patients with spinal muscular atrophy (SMA) type 2 or 3, according to a single-center ...

There is an urgent need for a national consensus and standardized care guidelines in Brazil to improve outcomes for patients with generalized pustular psoriasis (GPP), according to findings of a ...

A young man with worsening abdominal pain radiating to his back was diagnosed with IgG4-related retroperitoneal fibrosis (IgG-RRF). The report of this rare variation of immunoglobulin G4-related ...

Low-dose rituximab may offer transient efficacy in patients with autoimmune hemolytic anemia (AIHA), including those with cold agglutinin disease (CAD), according to a recent study published in ...

Tailored, risk-stratified rituximab regimens including yearly infusions may offer sustained disease control in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), ...

In patients with hepatocellular carcinoma (HCC), serum alpha-fetoprotein (AFP) levels are unreliable as a sole biomarker for disease diagnosis, according to findings from a retrospective study ...

A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) and received iptacopan, according to a study published in ...