This study aims to evaluate real-world effectiveness of pacritinib through a site-based retrospective chart review of medical records of adult patients with myelofibrosis treated with pacritinib in routine clinical settings with platelet count ≥50 x 10^9/L at the time of treatment initiation. Treatment effectiveness will be based on spleen size and symptom burden, hematological parameters, progression to leukemia, survival, pacritinib treatment patterns, patient demographic and clinical characteristics, and allogenic-hematopoietic stem cell transplant (allo-HSCT)-related outcomes. Data will be collected from medical charts of patients with pacritinib initiation between 01 June 2022 and 31 December 2025 with an end of study observation on 30 June 2026.

Iron deficiency is the most common nutritional deficiency worldwide and is a leading cause of anemia, which can result in symptoms such as fatigue, weakness, shortness of breath, and reduced quality of life. This study aims to better understand why some people develop iron deficiency by examining how the body absorbs iron and how this may relate to diet and proteins involved in iron absorption. Participants will complete questionnaires, provide blood, urine, and stool samples, undergo an oral iron absorption test, and complete a short food diary. If an upper endoscopy is already being performed as part of routine medical care, one additional biopsy sample will be collected during the procedure for research purposes. The information gathered may help improve the diagnosis and evaluation of iron deficiency anemia in the future. Participation involves two study visits over approximately one month. There is no direct medical benefit to participants, but the knowledge gained may benefit future patients with iron deficiency anemia.

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.

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.

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 study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms. 272 teens and adults with SCD will be enrolled in this study which is being conducted at the Medical University of South Carolina in Charleston SC., East Carolina University in Greenville NC., University of Miami in Miami FL., and the University of Alabama in Birmingham AL.

The purpose of this study is determine the optimal dose, efficacy and safety of an investigational drug (a new drug not yet approved by the U.S. Food and Drug Administration) in adults with Advanced Systemic Mastocytosis. The investigational drug is known as CGT9486 and will be taken daily orally. Participation in the study is expected to be approximately 6 years.

Over 2400 people who have sickle cell disease and are between the ages of 15 and 45 have been enrolled into the National Registry (SCDIC-I) of patients with Sickle Cell Disease (SCD). A rich resource of natural history data, the SCDIC-I Registry has longitudinal data collected yearly since 2016 from patient surveys (e.g. self reported pain incidences, sleep, barriers to care, experiences during and after pregnancy), medical record abstraction (e.g. medications, transfusion history, co-morbidities) and laboratory results. The 150 patients (or 1200 among the 8 sites) will be selected from both MUSC adult and pediatric SCD clinics starting at 12 years of age; those not previously enrolled in the SCDIC National Registry will be offered the possibility to enroll in SCDIC-II.
We will look at the following:
1- Compare the effect of new SCD medications – crizanlizumab, voxelotor, and L-glutamine – on clinical outcomes in individuals with SCD.
2 - Identify genetic and genomic predictors of response to crizanlizumab, voxelotor, and L-glutamine
3 - Integrate study data into the CureSCi metadata catalog (MDC) to enhance future cross-study analyses.

The purpose of this study is to see if taking depemokimab is safe and effective in treating Hypereosinophilic syndrome (HES) in adults (≥18 years) with uncontrolled HES receiving standard of care (SoC) therapy. The study will last approximately 52 weeks and is a placebo-controlled, double blind, multicentre study.

A device called the "Liposorber LA-15 System" has been approved by the
United States Food and Drug Administration for treating kids with focal
segmental glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used,
but the kidneys are still working okay. It can also be used if the subject
has had a kidney transplant and the FSGS comes back after the
transplant. Although the Liposorber System can be used for FSGS, we
are not sure how well the Liposorber System works. So, we are doing this
study to find out how well the treatment works in adults.
In this research study, there will be up to 5 adults who have FSGS
enrolled at MUSC. Subjects will come back for up to 12 treatments over 9
weeks and then 5 visits to their study doctor over the next 2 years.