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 ...
The D38A variant was the most frequent followed by the M13dup variant. Researchers identified 2 unique variants in the transthyretin (TTR) gene that are predominant among Korean patients with ...
The improvements occurred without any safety concerns, and the trial further indicated that higher total doses enable longer dosing intervals. Umedaptanib pegol, a novel anti-fibroblast growth factor ...
In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...
Bone marrow flow cytometry is one of the most efficient ways to diagnose or exclude PNH. A patient with a history of cortical vein thrombosis with new-onset pancytopenia was diagnosed with paroxysmal ...
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 ...
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 ...
Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...
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 ...
Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...
The novel anti-IL-36 receptor monoclonal antibody recibokibart demonstrated favorable safety and efficacy in treating patients with acute generalized pustular psoriasis (GPP) in a phase 1b study ...
Immunohistochemistry analyses detected a correlation between increased CDK1 expression and higher levels of LDH, a key indicator of greater disease severity in DLBCL. Higher levels of CDK1 expression ...