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Clinical Study

The HERO (Help Reduce organic Acids) Study

The HERO study is evaluating an experimental drug for methylmalonic acidemia (MMA) and propionic acidemia (PA). The study drug, called HST5040, is an oral therapy designed to correct metabolic abnormalities associated with MMA and PA. HERO will be conducted at select leading childrens hospitals in the United States. The HERO Study is recruiting participants age 2 and older with MMA or PA. The study is not enrolling patients who have had transplants at this time.


For more information contact:

Carrie Bailey

IRB#: IRB_00142396 | PI: Nicola Longo | Department: PEDIATRIC GENETICS | Approval Date: 2021-09-15 06:00:00
Specialties: Pediatric Genetics

Who can participate?

 Gender: All

 Age: All Ages

 Volunteers: Volunteers with special conditions

 Location: In Person

Will I be paid for my time?


Last Updated: 3/20/23