The JUMP (Journey to Understand MMA and PA) Study: A Natural History Study
Methylmalonic acidemia (MMA) and propionic acidemia (PA) are extremely rare conditions associated with significant morbidity and mortality. Because they are so rare, there is a great need to better understand these diseases to improve patient care and accelerate drug development. We have partnered with HemoShear Therapeutics on a study called the JUMP (Journey to Understand MMA and PA) Study to understand the clinical features, natural history and progression of MMA and PA over time. This poster describing the AllStripes platform, JUMP study design and pilot results of five patients was presented at the 43rd Annual Society for Inherited Metabolic Disorders (SIMD) meeting in April 2022.