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.

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.

This is a Phase 3, multicenter, randomized, parallel-group, double-blind, placebo-controlled study to evaluate the efficacy and safety of sonelokimab in adults with moderate to severe HS. Following a Screening Period of up to 28 days, each participant will enter a Placebo-controlled Period of 16 weeks (Part A) and subsequently a Crossover/Maintenance Period of 36 weeks (Part B). In Part A subjects will be randomly assigned in a 2:1 ratio to sonelokimab 120 mg or placebo. In Part B, participants who were initially randomized to placebo will cross over to sonelokimab and will receive this treatment for the remainder of the study. An End of Treatment (EOT) Visit will be performed at Week 52. After the EOT Visit, all participants who complete Week 52 will be offered the opportunity to enter an optional long-term open label extension (OLE) study under a separate protocol. For participants who do not progress to the OLE study, a Safety Follow-up Visit will be required 8 weeks after the last dose of study treatment.

The study includes both a retrospective cross-sectional and prospective longitudinal cohort study design. Demographic and clinical data will be obtained through medical record review. 3-4 blood draws will be collected over the course of the study. If participants were also in the MAC2v3 or NTM PRO Cohort study, data collected for that study will be used in this study. About 450 subjects are expected to participate in this study at 7 research sites in the United States. Participation in this study is expected to last up to 12 months. During that time, participants will have about 3-4 study visits.

This cross-sectional study aims to examine the impact of visual cues on listeners' perception of speaker age. Eligible speaking participants include adult males and females between the ages of 18–90 years old without voice or hearing disorders. Eligibility will be determined by research coordinators via patient demographic forms and validated voice and hearing assessments. Speaking participants will have two audio recordings taken: (1) sustained vowel sound and (2) reading of the Consensus Auditory-Perceptual Evaluation of Voice (CAPE-V), a standardized tool employed to evaluate the perceptual characteristics of voice quality across a wide age range. To develop the materials for the listening participants, CAPE-V audio recordings obtained from the speaking participants will be systematically paired with race and age-matched photographs obtained from a publicly available database. The finalized materials will encompass three experimental conditions: (1) audio only, (2) audio presented with a standardized photograph of an individual matched to the speaker's gender and age, and (3) audio presented with a standardized photograph of an individual matched for gender but differing in age. Naïve listening participants, blinded to the experimental conditions, will then be presented with these materials in a randomized order and asked to estimate the perceived ages of the speakers. Additionally, basic acoustic analyses of each sustained vowel sound recording (e.g., jitter, shimmer) will be conducted by a trained professional using the speech analysis software PRAAT.

The purpose of this research study is to assess if the study drug SION-719 is safe and well-tolerated in people with cystic fibrosis who are already taking Trikafta, understand the effect of SION-719 on the body by measuring chloride levels, and to learn how the body absorbs, distributes, and gets rid of the study drug in people with cystic fibrosis. This study is a placebo controlled cross-over study with two study treatment periods, and participants will take part in both study treatment periods. Participants will be assigned either study drug or placebo. Participants will remain on standard dose of physician-prescribed Trikafta throughout the study. This study will be approximately 16 weeks, or 112 days long.