The majority (79%) of cardiac amyloidosis cases were responsible for sudden cardiac death. Cardiac amyloidosis can be associated with sudden cardiac death and is often undiagnosed during a person’s ...

Large v-waves with an amplitude of 10 mm Hg or more were a determinant of all-cause mortality and rehospitalization due to heart failure events in patients with wild-type ATTR-CM. Elevated v-wave ...

The recommendations were based on the available evidence, its certainty, and key judgments using the Grading of Recommendations Assessment, Development and Evaluation framework. New guidelines ...

During this time, I was not on treatment for Pompe disease. When I was 26 years old, I got a new wheelchair. I had been using one for a long time, and the way I sat in the chair was not proper. I didn ...

There were 3 unmet needs in CIDP including the lack of specific biomarkers for the disease, weighing the relative value of various metrics, and understanding the optimal timing and approach to assess ...

Patients treated with deep brain stimulation of the centromedian nucleus (CM-DBS) showed improvements in seizure burden and alertness as reported by their caregivers. Deep brain stimulation of the ...

While current therapies for HAE are generally adequate, researchers continue to develop means to better alleviate the disease burden. To understand the direction of therapeutic development in ...

In the pivotal LUNA 3 trial, durable platelet response at week 25 was achieved by 23% of patients with rilzabrutinib compared with 0% with placebo. The US Food and Drug Administration (FDA) has ...

Intravenous immunoglobulin-induced thrombocytopenia was reported in 146 cases, with the highest incidence seen in patients with immune-mediated skin diseases. Intravenous immunoglobulin (IVIg) therapy ...

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

Rilzabrutinib significantly improved fatigue scores and reduced the need for rescue therapy by 52% vs placebo. Rilzabrutinib has shown clear evidence of efficacy and safety in patients with persistent ...

The 3 patients who underwent surgical gallstone removal all ultimately recovered well, including one who developed acute chest syndrome. As cholelithiasis and choledocholithiasis are common in ...