Histopathologic examination of a skin biopsy showed no features of psoriasis, suggesting hypocalcemia-related pustulosis after excluding drug-induced and inflammatory etiologies. Timely identification ...

This is the first report from SveATTR— a longitudinal Swedish web-based registry open for TTR variant carriers and patients with ATTR amyloidosis. The study summarizes 60 years of data collected as ...

Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...

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 ...

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

Kine Sciences advanced KINE-101 to the phase 1b/2a study following the successful completion of a phase 1 study in the United States. The first patient in a new phase 1b/2a clinical trial to ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

The RAPIDe-3 trial is evaluating an oral bradykinin B2 receptor antagonist in approximately 120 participants with hereditary angioedema. Pharvaris has updated its guidance, indicating that topline ...

PHOENIX, Arizona—Biomarkers “are a hot topic in myasthenia gravis (MG),” Anna Punga, MD, PhD, said during her keynote speech at the Myasthenia Gravis Foundation of America (MGFA) session of the 2023 ...

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 ...

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 ...

The levels of NT-proBNP, left ventricular mass index, maximal wall thickness, and E/E′ ratio were higher in patients with worse neurological stages compared to those in the early neurological stages ...