Are you an adult with Hereditary Hemorrhageic Telangiectasia (HHT) ? Information about people living with this rare genetic disease HHT is urgently needed. The purpose of this study is to collect a bank of information about people living with HHT to better understand HHT, the symptoms and complications it causes and how the disease impacts people's lives.

We are developing a computer-based tool for people who have cancers of the blood, bone marrow, and lymph nodes. This tool will help us understand what is most important to patients when it comes to the results of their treatment. We are looking for healthy volunteers (those without cancer) to help us test the usability of this tool.

The purpose of this study is to learn more about brain AVMs (BAVMs) and bleeding in HHT. We plan to study risk factors for rupture of BAVMs and also for other bleeding and features of HHT disease, including primarily genetics and imaging characteristics of the BAVMs. We hope that knowledge about risk factors will help improve the care and management of HHT patients.

To evaluate the safety of GBT021601 and its effect on sickle cell complications

Study the effectiveness and safety of Epeleuton

The primary objective of the study is to evaluate the safety and feasibility of treating patients with a history of iTTP with efgartigimod, an FcRn inhibitor that reduces IgG autoantibodies, including those responsible for ADAMTS13 depletion.

Purpose: Evaluate the efficacy of prophylactic Emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).

To have a well phenotyped cohort of people with sickle cell disease in order to examine risk factors for end organ disease through the creation of a multi-site registry and to develop a repository for the long-term storage of patient samples (whole blood, peripheral blood, serum) along with corresponding demographic and clinical information to allow for clinical and laboratory correlations.

This study is being done to answer the following question: Do two different anti-cancer drugs, paclitaxel (PTX) and pegylated liposomal doxorubicin (PLD) have similar effects on treating KS in PLWH in sub-Saharan Africa? We are doing this study because we want to find out if one of these approaches, PTX or PLD, is better than the other for KS. Both drugs may be given as part of the usual approach for your KS. The usual approach is defined as care most people get for KS.

We are surveying individuals with sickle cell disease (SCD) and/or parents of a child with SCD to get a sense of the types of treatment they would choose based on the harms and benefits of those treatment options. The proposed study will be designed to meet the following objectives: 1) Elicit individual respondent's maximum acceptable risk (MAR) for treatment benefits 2) Elicit individual respondent's minimum 3) Characterize heterogeneity in MARs and MABs among respondents