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

Autor: Thompson, William R., Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Campbell, John, Hornby, Brittany, Vaz, Frédéric M., Vernon, Hilary J.

Publikováno v: In Genetics in Medicine July 2024 26(7)

O07: Optimization of positive investigational outcomes in Barth syndrome using a combination of placebo-controlled, extended open-label, and natural history comparison studies*

Autor: Vernon, Hilary, Hornby, Brittany, Abbruscato, Anthony, Carr, Jim, Thompson, W. Reid

Publikováno v: In Genetics in Medicine Open 2023 1(1) Supplement

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ELAMIPRETIDE IMPROVES FUNCTIONAL ASSESSMENTS WHEN COMPARED TO THE NATURAL HISTORY PROGRESSION OF CARDIOMYOPATHY-RELATED DISEASE SYMPTOMATOLOGY IN PATIENTS WITH BARTH SYNDROME: A TAZPOWER ANALYSIS

Autor: Thompson, W. Reid, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Hornby, Brittany, Vernon, Hilary

Publikováno v: In Journal of the American College of Cardiology 11 May 2021 77(18) Supplement 1:575-575

Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial.

Autor: Karaa A; Massachusetts General Hospital, Genetics Division Harvard Medical School Boston, Boston, MA, USA. AKARAA@mgh.harvard.edu., Bertini E; Neuromuscular Unit, Bambino Gesù Ospedale Pediatrico, IRCCS, Rome, Italy., Carelli V; IRCCS Istituto Delle Scienze Neurologiche Di Bologna, Programma Di Neurogenetica, Bologna, Italy.; Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy., Cohen B; Akron Children's Hospital, Rebecca D. Considine Research Institute, Akron, OH, USA., Ennes GM; Stanford University School of Medicine, Stanford, CA, USA., Falk MJ; Division of Human Genetics, Department of Pediatrics, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Mitochondrial Medicine Frontier Program, Philadelphia, PA, USA., Goldstein A; Division of Human Genetics, Department of Pediatrics, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Mitochondrial Medicine Frontier Program, Philadelphia, PA, USA., Gorman G; Royal Victoria Infirmary, Newcastle Upon Tyne, England., Haas R; University of California, San Diego, La Jolla, CA, USA., Hirano M; Columbia University Irving Medical Center, New York, NY, USA., Klopstock T; Department of Neurology, LMU Hospital, Friedrich-Baur-Institute, Ludwig-Maximilians-Universität Munich, Munich, Germany.; German Center for Neurodegenerative Diseases (DZNE), Munich, Germany.; Munich Cluster for Systems Neurology (SyNergy), Munich, Germany., Koenig MK; Department of Pediatrics, Division of Child and Adolescent Neurology, Center for the Treatment of Pediatric Neurodegenerative Disease, University of Texas McGovern Medical School, Houston, TX, USA., Kornblum C; Department of Neurology, University Hospital of Bonn, Neuromuscular Diseases Section, Bonn, Germany., Lamperti C; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Lehman A; Vancouver General Hospital, Vancouver, BC, Canada., Longo N; University of Utah, Salt Lake City, UT, USA., Molnar MJ; Institute of Genomic Medicine and Rare Disorders, Semmelweis University, Budapest, Hungary., Parikh S; Cleveland Clinic Neurological Institute, Cleveland, OH, USA., Phan H; Rare Disease Research, Atlanta, GA, USA., Pitceathly RDS; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, London, UK.; NHS Highly Specialised Service for Rare Mitochondrial Disorders, Queen Square Centre for Neuromuscular Diseases, The National Hospital for Neurology and Neurosurgery, London, UK., Saneto R; Seattle Children's Hospital, Seattle, WA, USA., Scaglia F; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.; Texas Children's Hospital, Houston, TX, USA.; Joint BCM-CUHK Center of Medical Genetics, Prince of Wales Hospital, Sha Tin, Hong Kong SAR, China., Servidei S; Fondazione Policlinico Universitario A. Gemelli and Istituto Di Neurologia, Università Cattolica del Sacro Cuore, Rome, Italy., Tarnopolsky M; Division of Neuromuscular and Neurometabolic Disorders, McMaster University Children's Hospital, Hamilton, ON, Canada., Toscano A; Department of Clinical and Experimental Medicine, ERN-NMD Center for Neuromuscular Disorders of Messina, University of Messina, Messina, Italy., Van Hove JLK; University of Colorado and Children's Hospital Colorado, Aurora, CO, USA., Vissing J; Copenhagen Neuromuscular Center, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark., Vockley J; Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA., Finman JS; Jupiter Point Pharma Consulting, LLC, Jupiter, CT, USA., Abbruscato A; Stealth BioTherapeutics, Needham, MA, USA., Brown DA; Stealth BioTherapeutics, Needham, MA, USA., Sullivan A; Stealth BioTherapeutics, Needham, MA, USA., Shiffer JA; Write On Time Medical Communications, LLC, Medford, NJ, USA., Mancuso M; Department of Clinical and Experimental Medicine, Neurological Institute, University of Pisa, Pisa, Italy.

Publikováno v: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Nov 21; Vol. 19 (1), pp. 431. Date of Electronic Publication: 2024 Nov 21.