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 ...

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 ...

In the ADAPT SERON trial, treatment with efgartigimod led to rapid, significant, and sustained improvements in symptoms such as speech, vision, swallowing, and physical function. The US Food and Drug ...

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 ...

The prevalence of irregular red cell alloantibodies was 8.04% in pediatric patients, with anti-E and anti-Mi antibodies the most common. Pediatric patients can develop irregular, non-ABO red cell ...

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 ...

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 ...

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 ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...