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Patient and Trial Information

Kisha Jenkins, BS, RN

Forbes (Denny) Porter, MD

Latest News

Infant NPC Trial and Study Now Recruiting

Purpose of the Study:
The purpose of the study is to learn how well Adrabetadex can reduce liver damage in infants diagnosed with NPC disease.

Who will be enrolled?
Infants 6 months old or less with diagnosed NPC disease and evidence of liver disease are eligible to be screened for the trial.

What is involved if I participate?
The study has an initial 6-week phase during which the study participant will receive twice-weekly intravenous administration of the Adrabetadex drug. If there is evidence of clinical response, the participant is eligible to continue treatment for an additional 6 months.

Who is funding the study?
The National Institute of Child Health and Human Development of the National Institutes of Health

Who is the sponsor?
Patricia Dickson, MD

Who is the principal investigator (pi)?
Barbara Warner, MD

Where will the study take place?
St. Louis Children’s Hospital at Washington University Medical Center

Who do I contact for more information?
Patricia Dickson, MD
314-273-2943 |

Barbara Warner, MD
314-454-2683 |

Laura Linneman, RN
Research Nurse Coordinator
314-454-4950 |

More information can be found on



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