Results 161 to 163 of about 1,216,936 (163)

AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome

Human Gene Therapy, 2019
W Todd Cade, Barry J Byrne
exaly  

Barth Syndrome: Different Approaches to Diagnosis

Journal of Pediatrics, 2018
Masakazu Kohda, Atsuhito Takeda, Keiichi Hirono   +2 more
exaly  

Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER

Genetics in Medicine
Hilary Vernon, Frédéric Vaz, Brittany Hornby   +2 more
exaly