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 ...

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, ...

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 ...

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 ...

In this small cohort of adolescents with severe and/or refractory AAV, no drug-induced liver injuries nor opportunistic or serious infections were reported following treatment with avacopan. Currently ...

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 ...