Sabine Heine

TL;DR: The German PID-NET registry is a precious tool for physicians, researchers, the pharmaceutical industry, politicians, and ultimately the patients, for whom the outcomes will eventually lead to a more timely diagnosis and better treatment.

TL;DR: In this paper, age-related initial presenting manifestations of Inborn errors of immunity (IEI) including different IEI disease cohorts were analyzed. But, although increased infection susceptibility is a well-known initial IEI manifestation, less is known about the frequency of other presenting manifestations.

TL;DR: This study is designed to support haemophilia physicians using Emicizumab in physicians treating hemophilia and using (PWHAs) and with further post-marketing experience and trials, regular updates are necessary.

TL;DR: In this paper, the authors present an unabridged version of the interdisciplinary guideline for platelet disorders, which includes a stepwise algorithm for a rational diagnostic approach, and the PFA-100® CT is suited for screening for severe platelet defects.

TL;DR: A patient registry is made use of to characterize the burden of disease and the treatment modalities for patients with SCD in Germany.