Due to COVID-19, new enrollment in research studies will be greatly limited.
If you are interested in a study, there may be a longer waiting period to receive a response to your inquiry.
View the guidance for research participants for additional information.
The purpose of the study is to learn about the safety and efficacy of a medication called TransCon CNP in children ages 2-10 with achondroplasia. Children with achondroplasia lack this compound which is instrumental in promoting growth and ameliorating other potential medical problems. Read More
Children ages 2-10 (inclusive).
Have a clinical diagnosis of ACH with genetic confirmation
Have not entered puberty yet.
Able to stand without assistance.
Caregiver willing and able to administer subcutaneous (under the skin) injections of study drug.
This study enrolls adults and children ages 12+ who have obesity due to rare genetic disorders. The purpose of this study is to learn whether treatment with the study drug, setmelanotide, helps people with those disorders lose weight. Read More
Adults and children ages 12+.
Rare genetic disease patient with genetically confirmed diagnosis.
Obese (measured by BMI).
This study enrolls adults and children with Primary Immunodeficiency Disease (PID). The purpose of the study is to learn about the effectiveness and safety of a medication that may treat this condition. It is called Kedrion intravenous immunoglobulin (IVIg) 10%. Read More
-Adults and children ages 2-70.
-Diagnosed with PID and requiring treatment with IVIg.
-Documented agammaglobulinemia or hypogammaglobulinemia.