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Metabolism, Nutrition, and Physical Development Research Affinity Group Research Studies
1 - 8 of 8
Collaborative Registry for Williams Syndrome
CReWS (the Collaborative Registry for Williams Syndrome) has been developed for, and is owned by the Williams Syndrome Association.
Related Conditions:
This study is actively recruiting.
HRpQCT in Childhood Osteoporosis
A research study about bone health in children and adolescents at high risk of suffering a broken bone.
Related Conditions:
This study is actively recruiting.
Hunter Syndrome JR-141 Study
The purpose of this study is to find out if JR-141, an experimental drug, works better than the standard treatment, idursulfase, for MPS II (Hunter Syndrome) and how safe and effective it is in the management of central nervous system symptoms and other body symptoms related to MPS II.
Related Conditions:
This study is actively recruiting.
Lysosomal Storage Disease Registry
This research study is called the Lysosomal Storage Disease Registries.Related Conditions:
This study is actively recruiting.
MPS Biomarkers of Joint Disease
Establish connections between biomarkers and the stage and grade of bone and cartilage disease in MPS patients.
Related Conditions:
This study is not recruiting.
mRNA-3705 MMA Study
The purpose of this study is to find out more about mRNA-3705, a study drug that is being tested and is not approved by the U.Related Conditions:
This study is actively recruiting.
OT-58 for Homocystinuria
This research study is focused on patients who have Cystathionine beta synthase deficiency Homocystinuria (CBSDH).
Related Conditions:
This study is actively recruiting.
RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)
The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome).
Related Conditions:
This study is actively recruiting.