There was a reduction in volume of involvement, cardiac pyrophosphate activity, native T1, and extracellular volume during a median of 369 days between scans in patients with ATTR-CM being treated.

Patients diagnosed with wild-type ATTR-CM on or before 65 years of age had better survival after diagnosis compared to patients who were diagnosed after 65 years. Wild-type transthyretin-mediated ...

A multidisciplinary approach is necessary for the deep phenotyping and proper clinical management of individuals with primary mitochondrial diseases. A large percentage of patients (60%) with primary ...

Getty Images A post-hoc analysis of the ATTRibute-CM trial showed that acoramidis significantly improves survival in patients with p.V142I ATTRv-CM. By Month 30, the risk of combined all-cause ...

Results showed that 91% of patients had not been diagnosed or even suspected of having CA before death. Cardiac amyloidosis in the forms of transthyretin amyloid cardiomyopathy (ATTR-CM) and ...

Emerging therapies such as bezuclastinib and elenestinib show promise in improving disease control with potentially fewer toxicities. Other novel strategies, such as immune-based therapies, are under ...

Larry Luxner, senior correspondent for Rare Disease Advisor (RDA), interviews cardiologist Pradeep P.A. Mammen, MD, of the University of Kansas School of Medicine, on the growing awareness of girls ...

Unmanaged symptoms in hemophilia can restrict physical activity and reduce quality of life. Pharmacokinetic-guided treatment personalization allows patients with hemophilia A to become more physically ...

Health regulators in the US are leading a push away from animal models in preclinical studies of potential disease treatments. While animal models are likely to still have a place in translational ...

The highest in-hospital mortality and complication rates were found among Asian or Pacific Islander patients, and the longest hospital stays were found among African Americans and those classified as ...

The updated 2025 BSR management recommendations for patients with AAV provide systematic and evidence-based recommendations to support UK clinicians in managing AAV across the entire life course.

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