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 ...

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 ...

The physical environment was the only significant predictor of participation, explaining 34.1% of the variance. Children with Duchenne muscular dystrophy (DMD) participate less in daily life than do ...

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 ...

Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...

(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...

Generalized pustular psoriasis (GPP) is a rare and severe immune-mediated disorder in which widespread pustules form on the skin. 1 Pustules in GPP can affect large areas of the body, arms, and legs.

After some stumbles out of the gate, an experimental treatment that’s part of a group hoping to be the next big thing for people with multiple sclerosis (MS) is a step closer to US Food and Drug ...

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 ...

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 ...

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, talks with Claire Booth, MBBS, PhD, of University College London in the UK and cofounder of the AGORA Initiative, which aims to tackle the ...