The is a Phase 3 study for children, ages 0-17 years old, with severe von Willebrand disease (VWD). In this study, the study drug will be used prophylactically for the treatment of bleeding events. Prophylactic treatment means the study drug will be used to prevent or stop a bleed before it happens.
This study is going to look at how safe the study drug is and how well the study drug (recombinant von Willebrand factor (rVWF, vonicog alfa)), works to prevent and control bleeding.

The purpose of this rollover study is to investigate the long-term safety of etavopivat in participants11 months of age and older with SCD or thalassaemia who have completed a treatment period in previous etavopivat studies (parent studies, see Section 4.1). Long-term clinical efficacy measures of etavopivat treatment will also be assessed. This study will also ensure that participants who are benefiting from etavopivat treatment have prolonged access to the drug in the time before it is commercially available in their country.

This is a phase 2, randomized, multiple-dose, placebo-controlled, multicenter study to assess the safety, efficacy, and PK of CSL889 IV administration in adults and adolescents with SCD presenting with VOC. All subjects will receive a once daily dose of CSL889 or placebo until VOC resolution or Day 5, whichever comes first.

This is a study to test the safety of ALN-6400 which is considered to be "an investigational drug", meaning it is not currently approved by regulatory authorities, including the United States Food and Drug Administration (FDA), for the treatment of any disease and find out what effects, if any, it has on people with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB). ALN-6400 works by lowering protein in the blood breakdown blood clots and reduce bleeding in women ages 16-45. This study is a 24 week study with an optional extension period of 84 weeks.

The ATHNdataset Registry is the largest blood disorders real-world data set in the United States and is used to answer important clinical and scientific questions about the specific causes, prevention, treatment, and the social and economic impact of blood disorders.

The registry will involve collecting health information from the medical records, bleeding and treatment records of participants, and completion of questionnaires. This information will be part of the ATHNdataset registry which will be used to answer scientific, public health, and policy questions about better ways to treat blood disorders.