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 ...