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.

This is a cross-sectional, multi-center study comparing stool-based testing (multi-target DNA-based test and quantitative FIT test) to standard of care colonoscopy for CRC screening in PwCF. The study includes an enrollment visit, two stool samples completed at home, a clinical screening colonoscopy, and three participant surveys which will be completed at enrollment and remotely.
The total duration of participant participation will be 3-12 months, depending on the timing of the completion of the stool sample and the participant's colonoscopy. The targeted participant timeline is 3 months from enrollment to the completion of a clinical screening colonoscopy. To allow for varying site-specific clinical colonoscopy scheduling delays and COVID delays in scheduling, the duration of time between stool sampling and screening colonoscopy may extend up to 12 months for inclusion of data in the study. However, greater than 3 months duration between stool-based testing and colonoscopy will be considered a protocol deviation.

The study will be a multi-center, double-blind, randomised, placebo-controlled 2-period cross-over study in subjects with chronic cough due to idiopathic pulmonary fibrosis (IPF).
Subjects will participate in one of two cohorts (Cohort 1 and Cohort 2). Cohort 1 will evaluate a 30 mg orvepitant dose and Cohort 2 the 10 mg dose. Within each cohort, subjects will be randomised to receive either orvepitant or placebo in the first treatment period (Treatment Period A) followed by the alternate treatment in Treatment Period B. There will be a wash-out period of 3 weeks between the two treatment periods. Subjects will be randomised 1:1 to each of the two treatment orders and 1:1 to each cohort. Subjects will enter a screening period of between 14 and 28 days to determine eligibility. Eligible subjects will be randomised at the Baseline visit and will participate in two identical 28 day treatment periods with the wash-out period between them. There will be a total of 8 visits including the Screening, Baseline and final Follow-up visits.

Sometimes, it is necessary to re-learn a previously learned movement behavior, for example, a bad posture during the golf drive or while playing the piano. Unlearning or relearning an intensively trained behavior is particularly important if the behavior is hampering recovery, for example, in chronic pain or after a stroke. With this study, we experimentally test the brain mechanisms that control the change of pre-existing stable memories of a motor skill with electroencephalography (EEG). We will then use non-invasive brain stimulation to modulate these brain mechanisms and test if it is possible to change the pre-existing motor memory and the learning of a new motor skill.