Patients with ATTR-CM who experienced outpatient worsening heart failure had a 2.5-fold increased risk of mortality compared with those who did not. Outpatient worsening heart failure is an ...

Almost all (97%) of the patients with positive planar imaging and positive single-photon emission computed tomography findings were diagnosed with ATTR-CM. Single-photon emission computed tomography ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

ATTR-CM is a genetic condition characterized by the destabilization of transthyretin and subsequent misfolding and aggregation into amyloid fibrils that results in abnormal deposition of amyloid in ...

The diagnosis and management of CIDP are complex and best addressed by an interprofessional health care team that includes neurologists, neuromuscular specialists, physical medicine and rehabilitation ...

Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

Long-term vosoritide treatment produced sustained growth velocity increases of up to 2 cm/year over placebo in children with achondroplasia. Vosoritide, the first targeted pharmacologic therapy for ...

In a nonhuman primate model of acquired hemophilia A, treatment with PS-siRNA 40031 restored thrombin generation to normal levels. Reducing protein S (PS) levels can increase thrombin generation and ...

A systematic review and meta-analysis of trials in CIDP was published in 2023, studying the outcomes of patients including at least 2 years of follow-up. Studies conducted over 1 to 2 years ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...

Coramitug is well tolerated and leads to a statistically significant reduction in N-terminal pro-brain type natriuretic peptide (NT-proBNP)—a marker of disease progression in transthyretin-mediated ...