The purpose of this study is to test whether a drug called PRA023/MK7240 (the study drug) is a good treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD). The study drug PRA023/7240 is an investigational drug that is given by infusion every 4 weeks. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, PRA023/MK7240 will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of the PRA023/MK7240 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program and you will have 50% chance of receiving either the study drug or placebo. The study is double-blinded study and 50 weeks long, meaning you and your study doctor will not know what you are receiving, the study drug or placebo.

The study is sponsored by Prometheus Biosciences, Inc., a subsidiary of Merck & Co., Inc. The study is being done at approximately 25 sites across the United States. The main portion of the study will require 15 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys. If you complete the initial blinded treatment period of 50 weeks, the study doctor will discuss whether you are eligible to enter the open label period of the study, meaning no placebo. If you are eligible and agree, you will receive 500 mg of study drug once every 4 weeks for an additional 52 weeks. Compensation is available for participation.

This study is designed to evaluate a new therapy formulation for Alpha-1 Antitrypsin Deficiency (AATD). AATD is an inherited condition in which a person has low blood levels of a protein known as alpha-1 protease inhibitor (called Alpha1-PI). AATD causes an increased risk of chronic obstructive pulmonary disease (COPD) in the form of emphysema (long term lung disease) and, less frequently, other diseases.

This study is being conducted to evaluate the safety and tolerability of 2 different doses of Alpha-1 drugs (Alpha-1 15% and Liquid Alpha1-PI) in participants with AATD. Participants will be placed into one of two groups. Each group will receive both drugs at different points in the treatment period and because this is an "open label", study participants and the study staff know which dose of study drug participants receive. The study will last up to 486 days (16 months). Many visits are able to be conducted through home health care, lessening the need to come into the clinic.

Alpha-1 15% is an investigational product, meaning it is not approved by the U.S. Food and Drug Administration (FDA). The other drug in this study is Liquid Alpha1-PI (licensed as Prolastin®-C Liquid) and is an FDA approved treatment for adults with emphysema due to AATD. However, it is only approved for the recommended dose of 60 mg/kg. This study includes both the FDA approved 60mg/kg of Liquid Alpha1-PI and an experimental dose of 120 mg/kg that is not FDA approved. Alpha-1 15% is given as an injection under the skin and Liquid Alpha1-PI is given as an infusion into the veins.

You may or may not directly benefit from participation. However, you may help advance scientific knowledge in the treatment of AATD. Currently, the only FDA approved treatment for AATD is IV infusions of Liquid Alpha1-PI. Since the drug being studied, Alpha-1 15%, is injected with a small needle under your skin, there may be a benefit to future patients by providing flexibility of treatment route options as well as stability in serum alpha1-antitrypsin levels.

The purpose of this study is to test whether a drug called HZN-825 (the study drug) is an appropriate treatment for patients with Systemic Sclerosis (SSc). HZN-825 is an investigational drug that taken by mouth as a pill. An investigational drug is not approved by The US Food and Drug Administration and can only be used in a research study, like this one.

In this study, HZN-825 will be compared with a placebo (a pill that looks like the study pill but does not have any study drug in it). The comparison with the placebo helps to determine whether the effects seen in your body is because of HZN-825 or not. This is a randomized study meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. This will be done with the help of a computer-based program, and you will have 67% chance of receiving HZN-825. The study is double-blinded, meaning you and your study doctor will not know what you are receiving, the study drug or placebo. Should an emergency arise, and it is determined necessary, your study doctor will be able to find out what medication group you have been assigned.

The study is sponsored by Horizon Therapeutics Ireland DAC. The study is being done at approximately 135 sites across the world. The study is 60 weeks long will require 12 visits to the MUSC main campus. During study participation, the following procedures will be completed: blood draw, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High-Resolution Computed Tomography (HRCT)), electrocardiogram, as well as being asked to complete surveys about your overall health and wellbeing. Compensation is available for participation

The International Intestinal Failure Registry (IFR) is an initiative of the Intestinal Rehabilitation and Transplant Association (IRTA) and The Transplantation Society (TTS) and will be managed by these organizations. The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.

The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.

The purpose of this study is to test to see if you have a certain genetic mutation (changes in DNA) so we can learn more about kidney disease. The study involves one blood and saliva test and takes about 30 minutes. The blood test is to see if you have genetic changes in your DNA of a protein called APOL1. People who have this gene mutation may be at risk of losing their kidney function faster than others. The test won't cost you anything. In fact, if you decide to participate, you will be compensated $45. You should know that the test used to determine if you have an APOL1 genetic mutation is not FDA approved, however the FDA has approved this test for research purposes. If you were to participate in this study and take the blood test, and the result indicated you have this mutation, there may be an opportunity in the future to volunteer in an additional research study where you will receive the treatment. This treatment is designed by Vertex, especially for people with kidney disease from APOL1 mutation.

Individuals with alpha-1 antitrypsin (AAT) deficiency, emphysema and who have been enrolled in the SPARTA trial will be invited to participate in this study. This is a two year extension of the SPARTA trial for subjects who did not receive the study drug (Alpha 1 Proteinase Inhibitor) and for those who complete the SPARTA trial. Participants will all receive weekly infusions of Alpha-1 MP 60mg/kg either at MUSC or at home with a home health nurse. All participants will have blood work, pulmonary function test and CT scans done as part of this study. Safety and side effects of all therapies will be monitored.

The goal of this study is to develop an early systemic sclerosis (SSc) registry in the United States (US). A registry is a group of patients that are observed over time. This is a non-interventional study, meaning that they are no study specific medications to take or procedures to undergo. The specific aims include ongoing assessment of the natural history of early SSc patients by capturing and analyzing clinical data, patient reported outcomes, and laboratory data as seen in the clinic as part of their routine care needs. Additional study visits are not required. This is a multi-center study with sites spread across the U.S. This study is funded by the Scleroderma Research Foundation.

Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It results in the uncontrolled growth and proliferation of an atypical smooth muscle cells in the lung. These cells invade airways, blood vessels, and lymph vessels, and limit the flow of air, blood, and lymph, respectively. The source of the cells is unknown, but available evidence indicates they arise from an extrapulmonary source. Their aberrant behavior is due to mutations in tuberous sclerosis genes that results in mTOR activation. Respiratory failure, lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the disease. This study will evaluate the safety and durability of the mTOR inhibitors sirolimus and everolimus, which are FDA approved medications for prevention of rejection of transplanted organs, in stabilizing or improving lung function in people in LAM.

Individuals with alpha-1 antitrypsin (AAT) deficiency (AAT blood level lower than 11 micro-moles) and emphysema will be invited to participate in this study. This study will determine the impact of IV Alpha-1 proteinase inhibitor (Alpha-1 MP) on the progression of emphysema in patients with AAT deficiency. A participant in this study would receive any one of the following three therapies: 1) Alpha-1 MP dosed at 60mg/Kg, 2) Alpha-1 MP dosed at 120mg/Kg, or 3) Placebo. Once a subject is enrolled into this study, he/she will be randomly selected to receive only one of the above three therapies. Neither the study investigators nor the participants will know the actual therapy being given to the participants. All the study participants will receive serial chest CT scans to determine if their emphysema progresses over the following 3 years. Participants will have the IV therapies given to them weekly, with some infusions given at MUSC and some at home. Safety and side effects of all therapies will be monitored.

Systemic lupus erythematosus (lupus; SLE), Systemic Sclerosis (scleroderma; SSc) and Systemic Vasculitis (SV) are relatively rare rheumatic diseases that disproportionately impact the African American community, and particularly African American women. The causes of lupus, scleroderma and vasculitis are unknown, but thought to include both genetic and environmental factors. We are enrolling lupus, scleroderma, and vasculitis patients, and healthy control subjects. This is not a drug study. The purpose of this study is to better understand the factors that predispose people to develop lupus, scleroderma, and vasculitis. Information about medical, social and family history, medications, physical exam findings, and laboratory tests will be collected for analysis. This study will involve approximately 1360 volunteers.