Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

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 ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

Clonal dynamics varied across patients with advanced SM, with some subclones persisting despite treatment. Subclonal architecture analysis showed varying clonal dynamics, with some subclones ...

The navenibart phase 3 program will consist of the ALPHA-ORBIT phase 3 trial and the long-term extension trial, which are designed to support registration globally. Global start-up activities ...

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 ...

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 ...

(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) ...

In diagnosed cases of cutaneous mastocytosis, investigations to rule out systemic mastocytosis may be appropriate. A young child with a small chest lump and anemia was found to have cutaneous ...

Pediatric patients with ALL experience, on average, about 15 different symptoms in their chemotherapy journey. Pediatric patients with acute lymphoblastic leukemia (ALL) experience a wide range of ...

Yiannis Papazoglou is one of only 40 people in the world with a mutation of the IRF2BPL gene. Foundation Aims to Help Patients With Rare Diseases—Even Before They’re Recognized The Undiagnosed ...

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 ...