The purpose of this research study is to collect timely and relevant data that will support the evolving research needs of the Inherited Retinal Disease community (IRD), in order to provide insights that can be used to improve patient management, and to inform development of future treatments. No visits, assessments, or procedures are mandated, and follow-up will be captured as part of your standard of care. The planned length of registry is of 8 years with a potential to extend the duration as needs evolve.

The clear layer at the front of the eye that covers the pupil and iris (colored part of the eye) is called the “cornea”. When the cornea is damaged, it normally heals within a few days but it may take up to 2 weeks depending on the size and depth of the defect (wound). Some corneal defects heal much slower than expected. A defect in the cornea that fails to heal within the normal time of 2 weeks despite using the best available medicines and procedures, is known as Persistent Corneal Epithelial Defect (or PCED for short). The purpose of this research study is to evaluate the safety, tolerability, and effectiveness (risks and benefits) of of NEXAGON ophthalmic gel for the treatment of PCEDs.

We are exploring ways to promote healthy lifestyles during stroke rehabilitation using a web-based rehabilitation program. The purpose of this study is to compare two intervention approaches: teleABLE and Healthy Lifestyles Education. Both interventions are delivered using video visits, so participants can complete all study activities from home

This research is being done to determine if pectoserratus block with bupivacaine will provide improvement in pain control and decrease opioid use, relative to pectoserratus block with a saline solution, among patients undergoing total shoulder arthroplasty.

We are recruiting only healthy participants who do not have REM sleep behavior disorder. The information gained from this study will help the investigators understand more about REM sleep behavior disorder (RBD), and the possible underlying neurologic disorders that can cause RBD.

We hope to identify patterns in the brain that may be related to symptoms of PD, such as problems with walking. We are also testing different stimulation settings to see if they might be more effective than the settings that are currently used for treatment. This research may aid in our understanding of PD and how to treat it more effectively with DBS.

This protocol will characterize the effects of deep brain stimulation (DBS) location (both adverse and beneficial) on motor signs in people with Parkinson’s disease (PD). This information can be used to inform future DBS protocols to tailor stimulation to the specific needs of a patient. If targeted dorsal GP stimulation is shown to significantly improve motor features that are typically resistant to dopamine replacement therapy, these experiments will likely have major impact on clinical practice by providing a potential strategy to treat medically intractable symptoms.

This is a single center, open label, Phase I clinical trial of bioactive curcumin with high phenolic extra virgin olive oil (HP-EVOO) to treat cutaneous neurofibromas (cNF) in Neurofibromatosis, type 1 (NF1) patients (aged 18 years or older).

The purpose of this research to enhance our understanding of adrenoleukodystrophy ALD and study biospecimens such assaliva, blood, urine and stool to identify potential biomarkers for early identification of dise. We invite people who have or are at risk to have ALD, including females who are known or at risk carriers of the mutation for ALD, to help us learn more.

The purpose of the study is to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to iptacopan.

The purpose of this research is to better understand balance and walking in patients with Parkinson’s Disease. This research study uses small wearable devices that attach to clothing and can measure the body’s location while performing walking and balance tests.

The purpose of this study is to determine the dose of medication (Valacyclovir) needed to prevent an infant from developing herpes simplex virus (HSV) if the infant was potentially exposed to HSV at the time of delivery as they passed through the birth canal.

Researchers want to find out more about how physical, cognitive, and emotional factors affect eating.

The purpose of the trial is to evaluate the effect and safety of imlifidase when given to participants with antiGBM disease (also called Goodpasture disease). We will study if the addition of imlifidase to the standard of care treatment results in a better effect without causing unacceptable side effects compared to standard of care alone.

We are studying how well combination chemotherapy, bevacizumab, and/or atezolizumab work in treating people with deficient deoxyribonucleic acid (DNA) mismatch repair colorectal cancer that has spread from where it first started (primary site) to other places in the body (metastatic). Chemotherapy drugs, such as fluorouracil, oxaliplatin, and leucovorin calcium, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Bevacizumab may stop or slow colorectal cancer by blocking the growth of new blood vessels necessary for tumor growth. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving combination chemotherapy, bevacizumab, and atezolizumab may work better in treating patients with colorectal cancer.

We are looking at the addition of nivolumab to chemotherapy compared to usual treatment (chemotherapy alone) for the treatment of anal cancer that has spread to other places in the body. Immunotherapy, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as carboplatin and paclitaxel, work in different ways to stop the growth of tumor cells. Giving nivolumab with chemotherapy may help doctors find out if the treatment is better or the same as the usual approach.

We are studying how well trastuzumab emtansine (T-DM1) and tucatinib work in preventing breast cancer from coming back (relapsing) in patients with high risk, HER2 positive breast cancer. Trastuzumab is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as HER2 receptors, and delivers DM1 to kill them. Tucatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

The purpose of this study is to compare the effects of 2 different levels of exercise intensity and to learn more about effects of aerobic exercise for people with Parkinson’s disease (PD). This study will help us better understand what exercise guidelines should be used in the future.

Participants who take part in this study will receive a study drug called ruxolitinib with a standard drug called nivolumab. The study is being done to measure the percentage of tumor (lymphoma) that shrinks after receiving ruxolitinib in combination with nivolumab. This study will also measure the length of time the lymphoma is inactive and how safe the combination is to administer to participants. Ruxolitinib is a pill that is taken twice every day. Nivolumab is given as an infusion in the clinic once every 4 weeks.

The purpose of this study is to capture clinical information on how kidney disease affects the health of women, focusing on menstrual and reproductive health, the transition to menopause (the time when your menstrual periods stop permanently), and menopause itself. We also wish to learn more about how women’s health affects their kidney disease, bone and heart health.

The purpose of this research study is to learn more about how food affects blood sugar levels, and whether a continuous glucose monitor (CGM) can help to individualize nutrition education for people with diabetes. Participation in the study would require 3-4 clinic visits over a period of 14 weeks. Participants will also be asked to: meet with a registered dietitian every 2 weeks (virtually), keep food logs, wear a CGM and an activity monitor, answer survey questions, and provide blood samples to measure markers of diabetes control (like hemoglobin A1c).

To determine the prognostic factors for visual outcome for newly diagnosed NF1-OPGs. Hypothesis: Patients (<18 years of age) with tumors involving the optic tracts and/or radiations will demonstrate worse visual outcomes compared to those without optic tract involvement.

This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

The purpose of this multicenter study is to compare outcomes between patients who will undergo different types of ACL reconstruction. All patients will have a tendon from their own knee used to reconstruct the ACL. Prior to knee surgery, researchers will randomize (i.e. a coin toss) to determine if ACL reconstruction will be done with patellar or quadriceps tendon and if the lateral extra-articular tenodesis will or will not be added to the ACL surgery. We will follow-up with participants as they undergo treatment and recovery after surgery for 2 years.

PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.

The purpose of this study is to look at a different type of electroconvulsive therapy (ECT) that may reduce negative side effects while still providing relief from symptoms of major depression.

The purpose of this pilot research study is to test whether a tool called “High-Resolution Manometry” can diagnose laryngeal dystonia (also known as spasmodic dysphonia) and measure how well treatment works. High-Resolution Manometry measures pressures from a small catheter that is passed from your nose into your throat. We believe that pressures in the throat might be different for people with laryngeal dystonia than for people without laryngeal dystonia, or with other types of voice disorders. If we can diagnose laryngeal dystonia shortly after symptoms start, we can get patients the treatment they need sooner.

More than 65% of people with lower extremity peripheral artery disease (PAD) are overweight or obese. Overweight or obese people with PAD have greater functional impairment and faster functional decline than normal weight people with PAD. Walking exercise is first line therapy to improve walking performance in PAD. However, our NHLBI-funded observational longitudinal study of functional decline in PAD showed that overweight and obese PAD participants who combined weight loss with walking exercise had significantly less functional decline than those who walked for exercise but did not lose weight. Therefore, we hypothesize that among people with PAD who are overweight or obese, a weight loss intervention combined with exercise will improve walking ability more than exercise alone. However, the effects of intentional weight loss in overweight or obese people with PAD are unknown and may not be beneficial if weight loss exacerbates PAD-related sarcopenia. Behavior change that achieves sustained weight loss is particularly challenging in older obese people with chronic disease. Therefore, among people with PAD and BMI>28 kg/m2, we will conduct a randomized clinical trial to test the hypothesis that a weight loss intervention combined with walking exercise achieves greater improvement in functional performance than exercise alone at 12-month follow-up.

This is a study about how to recruit sexual and gender diverse people into cancer research studies. You will be asked to participate in a secure Zoom interview; if you do not have access to Zoom an interview may occur over the phone. The interview will take about 60-90 minutes, and we will ask questions about your experience with participating in cancer research studies and your experience during treatment.

The purpose of this study is to evaluate the long-term safety and effectiveness of Skytrofa treatment in children growth hormone deficiency. Patient care will follow the normal treatment practices at the clinic. No additional visits will be performed beyond the usual clinical practice.