The pathogenic role of CD38-expressing plasma cells in FSGS may be linked to the therapeutic effect, given that circulating CD38+ plasma cell counts have been shown to correlate with disease activity.

Recurrent atraumatic vertebral fractures warrant serum tryptase, KIT testing, and biopsy at augmentation. Indolent systemic mastocytosis (SM) should be suspected in patients presenting with atraumatic ...

Best practices most often mentioned by amyloidosis stakeholders included patient-centric multidisciplinary care, responsiveness to patients' questions, and timely intake of new patients who are at ...

The FDA granted Fast Track Designation to coramitug for ATTR-CM as it advances in the ongoing phase 3 CLEOPATTRA trial. The US Food and Drug Administration (FDA) has granted Fast Track Designation to ...

The adjusted mean change in forced vital capacity at week 52 was –107.1 mL in patients given placebo, –61.2 mL in patients treated with 9 mg of nerandomilast twice a day, and –64.9 mL in patients ...

At week 52, 50.9% of patients on elafibranor met the primary endpoint of biochemical response compared with 3.8% of those receiving the placebo. Lower baseline alkaline phosphatase (ALP) levels were ...

There were 7 cases of PAH temporally associated with bevacizumab in the French Pulmonary Hypertension Registry, but most had predisposing conditions. Bevacizumab exposure does not seem to lead to the ...

Researchers developed an AI-based framework that can generate a report like a primary care physician, which can then help identify undiagnosed patients with ATTR-CM and individuals who are at risk of ...

Test Your Knowledge About the Signs and Symptoms of FNAIT Test Your Knowledge About the Epidemiology of FNAIT Test Your Knowledge About the Diagnosis of FNAIT Rare Disease Advisor, a trusted source of ...

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 ...

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 ...