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 ...

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

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 potential of axonal damage as a biomarker of disease severity and predictor of further outcomes is highlighted by its effect on functional disability, even in early disease stages. Patients who ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

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 ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...

My recent liver biopsy was a case study of Murphy’s Law in action. And as I share the details of my saga, I’m mindful that my experience is the exception, not the rule. I do not want to discourage ...

Both a higher Charlson Comorbidity Index score and older age showed a significant prognostic impact on all-cause mortality. Chronic lymphocytic leukemia (CLL) progression was the leading cause of ...

(L-R) Durhane Wong-Rieger, Cynthia Cassandro, Jida El Hajjar, Armelle Pindon and Richie Kahn participate in a panel discussion at the 2024 World Orphan Drug Congress USA. (Photo by Manuela Callari, ...

Almost all patients with CIDP taking azathioprine (8 of the 9 patients) went into remission. Oral immunosuppressive drugs, such as azathioprine, may be a cheaper and more convenient alternative or ...