The extracellular volume on cardiac MRI was markedly higher in patients with PyPn-negative ATTR-CM compared to controls. The extracellular volume derived from cardiac magnetic resonance imaging (MRI) ...

Thoracolumbar kyphosis angle improved across age groups, with fewer children treated with vosoritide exceeding 20° at week 52 (33% vs 57%). Vosoritide, a C-type natriuretic peptide analog that ...

The Nakanuma bile duct loss score may be an important indicator for the prognosis of PBC in the early stages of the disease, a study found.

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 ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

This unique case underscores the importance of meticulous prenatal and postnatal immunologic workup when alloimmune conditions are suspected. A 37-year-old multiparous woman with RhD alloimmunization ...

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

A patient presented with task-related isolated transient weakness in her right hand, which started 15 years before and lasted for 9 years without worsening or improvement. Distal myasthenia gravis (MG ...

Obeticholic acid, seladelpar, and elafibranor all significantly increased the biochemical response rate. Elafibranor seems to be slightly more effective than seladelpar as a second-line therapy in ...

The researchers used the Paris criteria to reach a final diagnosis. A team of international researchers presented the rare case of a male patient with autoimmune hepatitis-primary biliary cholangitis ...

Hemolysis ceased completely in 2 patients after receiving 20 mg of rituximab. Low-dose rituximab may offer transient efficacy in patients with autoimmune hemolytic anemia (AIHA), including those with ...

The primary goals of AI personalization were to improve onboarding, boost medication initiation and adherence, and enhance physician follow-up. AllazoHealth’s artificial intelligence (AI)-powered ...