The Tp-e interval, and Tp-e/QT Tp-e/QTc ratios were significantly prolonged in patients with wild-type ATTR-CM compared to healthy people. Electrocardiographic (ECG) parameters are prolonged in ...

Spesolimab is a humanized monoclonal immunoglobulin G1 antibody and is the first therapy to target the IL-36 pathway, a key driver of GPP disease processes. Spesolimab has an acceptable safety profile ...

Inebilizumab-cdon (Uplizna ®) has received US Food and Drug Administration (FDA) approval for the treatment of adults with generalized myasthenia gravis (MG) with antibodies against the acetylcholine ...

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 ...

Biochemical response rates were sustained through week 182 in patients with PBC treated with elafibranor, and the proportion of patients with normal alkaline phosphatase levels remained consistent.

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 ...

Lonvoguran ziclumeran, an experimental gene editing therapy for HAE, is known as lonvo-z. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Catherine Miller, PharmD, global ...

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 ...

Nipocalimab is currently being evaluated in 2 international randomized studies for HDFN: the UNITY and AZALEA trials. With nearly 1000 times greater affinity for FcRn than intravenous immunoglobulin, ...

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 ...

The results of the current meta-analysis reinforce the safety and efficacy of marstacimab in reducing annualized bleeding rates among patients with severe hemophilia A and B. Among patients with ...

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 ...