Quality of life in Barth syndrome.
Alexander Y KimHilary VernonRyan ManuelMohammed AlmuqbilBrittany DeCroesPublished in: Therapeutic advances in rare disease (2022)
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects with Barth Syndrome (TAZPOWER). https://clinicaltrials.gov/ct2/show/NCT03098797.Registration Number: NCT03098797.