This study looks at understanding eating behaviors and attitudes toward food in children/adolescents. More specifically the study's goal is to compare those with and without food allergies to gain a better understanding of a possible underlying factor towards certain behaviors. Children and their caregivers will complete a survey composed of a short section on the child's medical history regarding allergies followed with questions in commonly used clinical assessments.

This research study of fazirsiran (FAZ-i-sir-an; also called TAK-999 or the “study drug”) because you have alpha-1 antitrypsin deficiency-associated liver disease (AATD LD) with METAVIR stage F2 to F4 fibrosis (METAVIR is a system used to score the amount of inflammation and fibrosis seen in a liver biopsy). In AATD, abnormal (Z-AAT) proteins build up in liver cells, leading to varying amounts of liver problems. The goal of treatment with fazirsiran is to prevent and improve the build up of these abnormal proteins that cause liver injury and fibrosis

-The total amount of time you may be involved in the study is about 4 ½ years (230 weeks). -Screening Period of up to 70 days. -Treatment Period of 196 weeks or about 4 years. During the study, you will get the study drug or placebo at the study site. -Follow-up Period of 6 months. You will have visits 6, 12, and 24 weeks after your last injection of the study drug or placebo. -You will have lung function tests (PFT and DLCO) to check how your lungs are working At every visit during the treatment period you will have: -Your vital signs will be measured, this includes your heart rate, blood pressure, breathing rate, temperature, and amount of oxygen in your blood. -You will have a brief physical exam. -Your weight will be measured. -You will have an ECG. -Collect lab samples During specific study visits during the treatment period the following tests will be performed: -An abdominal ultrasound -FibroScan -CT scans You will complete questionnaires.

This study will investigate the effects of NTLA-2002 on people with Hereditary Angioedema (HAE). NTLA-2002, which consists of a CRISPR/Cas9 gene editing system, is designed to limit production of plasma kallikrein in the liver cells by acting on the KLKB1 gene. This means NTLA-2002 would permanently change the DNA in the liver cells so less plasma kallikrein would be produced. Lower levels of plasma kallikrein may result in fewer HAE attacks.

During part 1 of the study, participants will either be given NTLA-2002 or placebo (no active ingredients) as an intravenous infusion. Participants will have the option to receive the opposite infusion about 28 weeks later. This study involves getting pre-medications to reduce the chance of an HAE attack and a reaction to the infusion. Study assessments will be preformed to monitor safety. Daily electronic diaries will be completed to monitor for HAE attacks. Also quality of life questionnaires will be completed. If part 1 and part 2 are completed, there are 22 visits. Some of those visits can occur over the phone.

A clinical research study called EMBARQ-CSU, which seeks to assess an investigational study medicine that has the potential to expand treatment options for Chronic Spontaneous Urticaria (CSU). CSU is a skin condition also known as chronic hives or long-term hives that is characterized by raised itchy welts on the skin that persist for six weeks or longer with no known cause.

If you decide to take part in this research study, you will have a screening phase where you will have procedures to determine if you meet the requirements to be in the study. These include a physical exam and an electrocardiogram (ECG) which will measure the electrical activity of your heart. You will also be required to give blood and urine samples. If you are a woman who can have children, you will also need to take pregnancy tests. If you qualify and agree to take part in the study, you will receive injections of barzolvolimab or placebo (the placebo contains the same ingredients but without barzolvolimab) under your skin every 4 weeks for 6 months. After 6 months, everyone will receive barzolvolimab for an additional 6 months, involving visits every 4 weeks. During the study, you will continue to have physical exams, blood taken for testing, and urine tests. You will need to complete a daily diary.

This is a voluntary clinical research study to investigate BW-20805 a injectable treatment for hereditary angioedema (HAE). The purposes of the study are to evaluate the safety and efficacy of the study drug for the treatment of HAE, the effectiveness of the study drug for preventing HAE attacks, and how the study drug works in the body and the relationship between the amount of the study drug taken and the effect on the body.

You will answer questions about your health, provide blood samples, undergo routine urine laboratory tests, receive the study drug, undergo electrocardiogram (ECG), complete questionnaires, undergo physical exams, and provide HAE attack information. There are a total of 13 visits.

The purpose of this study is to evaluate the effectiveness and safety of navenibart as a preventative treatment for people with Type I or Type II Hereditary Angioedema (HAE).

You will need to come to Penn State Health Milton S. Hershey Medical Center 8 times. You will answer questions about your health/HAE attacks, provide blood samples, undergo routine urine laboratory tests, receive the trial drug, undergo electrocardiogram (ECG), complete questionnaires, undergo physical exams, and provide HAE attack information.

The purpose of this clinical research study is to learn more about the use of the investigational drug, deucrictibant, for the prevention of HAE attacks. You will either receive 40mg deucrictibant or a placebo tablet every day for 24 weeks. The study drug is a tablet that you will take by mouth. Every day you will have to take 1 tablet with a glass of water, at approximately the same time each day either without a meal or with a light meal. The study consists of a Screening Phase which may last up to 10 weeks, a Treatment Phase (24 weeks), and a Follow-up Phase (up to 4 weeks). These are all described below. If you provide your consent to take part in the study and you are declared eligible to participate, your overall study participation will last approximately 10 months (38 weeks).

There will be approximately eight in person visits, blood will be drawn at six of these visits, an EKG will be performed at five of these visits, vital signs will be performed at seven of these visits, and a physical exam will be performed at five of these visits. You will be required to complete questionnaires at each visit, and complete an electronic diary.

The purpose of this clinical research study is to learn more about the use of the investigational drug, deucrictibant, for the prevention and treatment of Acquired Angioedema due to C1 Inhibitor Deficiency (AAE-C1INH) attacks. The study consists of a Screening Phase (up to 10 weeks), followed by a 12-weeks Prophylaxis Treatment Phase (Part 1), an On-demand Treatment Phase (Part 2) and an Open-Label On-demand Treatment Phase (Part 3). These are all described below. If you provide your consent to take part in the study and you are declared eligible to participate, your overall study participation will last approximately 15 months (60 weeks). During this study you will be asked to complete an electronic diary daily. At the study visits you will have blood samples collected. Vital signs, EKGs, and physical exams will be performed.

If you decide to take part in this research study, you will have a screening phase (up to 10 weeks), followed by a 12-week Prophylaxis Treatment Phase (Part 1), an On-demand Treatment Phase (Part 2) and an Open-Label On-demand Treatment Phase (Part 3). If you provide your consent to take part in the study and you are declared eligible to participate, your overall study participation will last approximately 15 months (60 weeks). where you will have procedures to determine if you meet the requirements to be in the study. These include a physical exam and an electrocardiogram (ECG) which will measure the electrical activity of your heart. You will also be required to give blood and urine samples. You will also need to complete a daily electronic diary. If you are a woman who can have children, you will also need to take pregnancy tests.

This research study is being done to see how well the Study Drug, also called donidalorsen (ISIS 721744), works and if it is safe for children with HAE age 2 to less than 12 years old. Donidalorsen is approved in the United States to prevent HAE attacks in adolescent and adult patients 12 years of age or older. The Study Drug is designed to lower the amount of a protein, known as prekallikrein (PKK), which is made by the liver. When PKK is made by the liver and released into the blood stream, it can lead to HAE attacks. Therefore, the purpose of this study is to assess if reducing the amount of PKK can reduce your child’s HAE attacks and to assess the safety of donidalorsen for children with HAE age 2 to less than 12 years old. This study is an open-label study which means that your child and all participants enrolled in the study will receive active Study Drug. Study participants will receive a subcutaneous (under the skin) injection of donidalorsen once every 4 weeks. The Study Drug dose (10, 40 or 80 mg) will be determined by your child’s body weight and may change during the study. Following screening, you and your child will be asked to come to the study site for 14 visits over about 1 year during the initial treatment period. There is also a Post-Treatment Period your child may choose to enter, which requires 2 phone calls and 1 final visit over 13 additional weeks.

This study is an open-label study which means that your child and all participants enrolled in the study will receive active Study Drug. Study participants will receive a subcutaneous (under the skin) injection of donidalorsen once every 4 weeks. The Study Drug dose (10, 40 or 80 mg) will be determined by your child’s body weight and may change during the study. Following screening, you and your child will be asked to come to the study site for 14 visits over about 1 year during the initial treatment period. There is also a Post-Treatment Period your child may choose to enter, which requires 2 phone calls and 1 final visit over 13 additional weeks. During the Study Treatment and Post-Study Treatment Periods, there is also the option to have some of your child’s visits done by a home health care nurse. At each study visit, height and weight will be collected. A physical exam will be performed. An ECG and vital signs will be collected, paper questionnaires will need to be completed, you and/or child will be asked questions about your child's health, medications and medical history. Any HAE attacks will need to be reported to the study staff.