You or your child are being asked to take part in this research study because you have Glycogen Storage Disease Type III (GSD III). The purpose of this research study is to better understand blood sugar variability, muscle strength and function, and impact of Glycogen Storage Disease Type III (GSD III) on overall health. You will not receive any treatment for your GSD III as part of this study, but the information you provide will be helpful for the study sponsor, Ultragenyx Pharmaceutical Inc., to develop a new therapy for GSD III.

If you agree to take part, your participation will last for about 26 weeks and will involve up to 2 study visits to the hospital along with up to 26 phone calls. There will be no changes to your treatment regimen for GSD III as part of this study.

As a participant in the research you will:

• come to the hospital for 1-2 in person visits to complete interviews and questionnaires,
• wear a blinded continuous glucose monitor (CGM) 3 months and an unblinded CGM for 3 months,
• complete fingerstick blood sugar checks, maintain a nutrition diary for 3 or more weeks throughout the study,
• maintain a hypoglycemia symptom diary, and
• upload CGM and HHG device data to a study-provided laptop weekly.

The main risks of this study are from hypoglycemia related to your diagnosis of GSD III and a loss of confidentiality. There are no direct benefits to you for participating in this study but the information collected as part of this study will help researchers better understand GSD III.

Our team is working on a clinical drug trial that involves giving you a study drug that is not FDA-approved called RZ358. The goal of the trial is to test the study drug safety, how your body processes the drug, how the drug affects your body, and see if it can increase your blood sugar. If you agree to take part, your participation will last for up to 27 weeks and include 4 weeks of screening, 8 weeks of treatment, and a 15 week follow-up period. It is possible that treatment with the study drug infusions may improve your blood sugar levels and/or reduce the use of other medication(s) to prevent hypoglycemia. For more information, contact us at HIResearch@email.chop.edu.

The purpose of this study is to find out if an investigational drug called setmelanotide (RM-493) can help control body weight in people with certain genetic modifications in their genes that play a key role in the regulation of body weight. Males and females, age 6 to 65, with obesity and certain genetic variants (MC4R pathway) may be eligible to participate. If you are confirmed eligible for the study, then participation will last up to 64 Weeks. Participants will complete Screening (Up to 8 Weeks), Double-Blind Treatment (Up to 52 Weeks), and Follow-Up Period (4 Weeks). Participants will complete research procedures including physical exams, blood draws, ECGs, and questionnaires. Participant will also need to self-inject the study drug once daily for the duration of their participation.

We are currently enrolling adolescents ages 15-21 of South Asian ancestry, African American ancestry, and European American or "White" ancestry. This study is looking to better understand ancestry related differences in risks for developing cardiovascular disease and type 2 diabetes. The study involves 2 visits to the CHOP Main Hospital in Philadelphia, and participants will be compensated for their efforts.

Insulin therapy, commonly given as multiple daily injection therapy, is the only recommended treatment for Cystic Fibrosis Related Diabetes (CFRD). Traditional therapy for CFRD requires an intense daily effort related to diabetes care on top of the already burdensome management of Cystic Fibrosis. In this study, we will assess the safety and effectiveness of the iLet Bionic Pancreas, an automated insulin delivery system, which uses a continuous glucose monitor (CGM), an insulin pump, and a control formula that activates insulin delivery based on CGM glucose data.