Researchers recently detailed the genetic and clinical features of congenital protein-losing enteropathy (PLE), with a focus on CHAPLE disease.

The TibuSURE trial will assess tibulizumab, a dual-antagonist antibody designed to target IL-17A and B-cell activating factor. This is the first time it is being considered as a therapeutic option in ...

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 ...

A chronic, nonproductive cough is another major presenting symptom of patients with IPF. As many as 80% of patients with IPF experience chronic cough. Chronic cough usually lasts at least 8 weeks and ...

Segmental analysis showed significant correlations between 3D-STE and CMRI strain measurements across most regions. Two advanced echocardiography techniques reliably detect both global and regional ...

Progressive pulmonary fibrosis (PPF) is defined as interstitial lung disease (ILD) characterized by worsening respiratory symptoms, lung function decline, and continuing fibrosis. 1 As the disease ...

Delivered as a single intracerebroventricular injection, the therapy is designed to provide long-term benefit and potentially address the full range of DS symptoms. ETX101, an investigational gene ...

Doctors should closely monitor patients with GPP for signs of PG, including a thorough assessment of painful ulcers of undetermined origin. A diagnosis of generalized pustular psoriasis (GPP) is ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

ATTR-CM is a genetic condition characterized by the destabilization of transthyretin and subsequent misfolding and aggregation into amyloid fibrils that results in abnormal deposition of amyloid in ...

The earliest descriptions of symptoms consistent with IPF were reported by German physicians in the late 1800s and early 1900s. The disease became much more widely known following English-language ...

Regional analysis showed the highest 2022 mortality in the Americas (1.80 per 100,000), followed by the Western Pacific (1.21) and Europe (1.11). Global mortality from idiopathic pulmonary fibrosis ...