The global prevalence of PNH is as high as 15.9 individuals per 1 million people. The annual global incidence of PNH is around 5 to 6 individuals per 1 million people. 5,6 In the United States, ...

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 ...

As of May 2025, 62 active clinical trials are ongoing for Fabry disease, including 25 interventional and 37 observational studies, of which 8 are patient registries. Among the trials with known ...

Oculomotor abnormalities and respiratory insufficiency—a feature shared with TK2d—appeared to be less frequent than previously described. Researchers identified 28 new cases of NDUFV1-related disease, ...

Claseprubart incorporates YTE half-life extension technology, which is designed to enable infrequent subcutaneous self-administration. A phase 3 study is underway to evaluate the investigational ...

The researchers observed that the specific assessment method heavily influenced the prevalence estimates. Anxiety and depression are common among patients with idiopathic pulmonary fibrosis (IPF), and ...

IPF, the most common form of fibrosing interstitial lung disease (ILD), is characterized by abnormal accumulation of fibrotic tissue, causing thickening and stiffening of the lung parenchyma. 2 ...