Patients with ATTR-CM had significantly impaired global longitudinal strain and higher mechanical dispersion compared to their first-degree relatives who were not affected by the disease. Increased ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease (HD) since 1969. Dr. Lange, a proponent of nondrug ...

New research indicates that obinutuzumab is a potentially effective and cost-effective treatment for patients with rituximab-resistant AAV. Obinutuzumab therapy demonstrated immunological superiority ...

US and European regulators could approve up to 17 gene therapies this year, with a top official at the US Food and Drug Administration (FDA) predicting that 2024 will be a “breakout year” in ...

Jennifer Miller, MD, a professor of pediatric endocrinology, speaks about her research on Prader-Willi syndrome from her office at the University of Florida in Gainesville. (Photo by Larry Luxner) ...

Kine Sciences advanced KINE-101 to the phase 1b/2a study following the successful completion of a phase 1 study in the United States. The first patient in a new phase 1b/2a clinical trial to ...

Immunohistochemistry, immune-electron microscopy, and proteomic analyses can be used for amyloid typing. Endomyocardial biopsy is the gold standard for the diagnosis and characterization of cardiac ...

Intravenously administered zaltenibart was shown to be safe and effective in phase 2 clinical trials. Enrollment is under way to recruit patients for a phase 3 study designed to assess intravascular ...

The Muldoon family from Philadelphia, Pennsylvania, is shown after baby KJ’s infusion of a personalized gene-editing therapy (Photo courtesy of Children’s Hospital of Philadelphia) KJ Muldoon has been ...

Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...