Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome
Hornby, Brittany, Thompson, William Reid, Almuqbil, Mohammed, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Vernon, Hilary J
Published in Orphanet journal of rare diseases (02.09.2022)
Published in Orphanet journal of rare diseases (02.09.2022)
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Journal Article
O07: Optimization of positive investigational outcomes in Barth syndrome using a combination of placebo-controlled, extended open-label, and natural history comparison studies
Vernon, Hilary, Hornby, Brittany, Abbruscato, Anthony, Carr, Jim, Thompson, W. Reid
Published in Genetics in Medicine Open (2023)
Published in Genetics in Medicine Open (2023)
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Journal Article
ELAMIPRETIDE IMPROVES FUNCTIONAL ASSESSMENTS WHEN COMPARED TO THE NATURAL HISTORY PROGRESSION OF CARDIOMYOPATHY-RELATED DISEASE SYMPTOMATOLOGY IN PATIENTS WITH BARTH SYNDROME: A TAZPOWER ANALYSIS
Thompson, W. Reid, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Hornby, Brittany, Vernon, Hilary
Published in Journal of the American College of Cardiology (11.05.2021)
Published in Journal of the American College of Cardiology (11.05.2021)
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Journal Article
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Thompson, William R., Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Campbell, John, Hornby, Brittany, Vaz, Frédéric M., Vernon, Hilary J.
Published in Genetics in medicine (01.07.2024)
Published in Genetics in medicine (01.07.2024)
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Journal Article
Safety, pharmacokinetics and pharmacodynamics of SBT‐020 in patients with early stage Huntington's disease, a 2‐part study
Diemen, Marcus P.J., Hart, Ellen P., Abbruscato, Anthony, Mead, Liz, Beelen, Ilse, Bergheanu, Sandrin C., Hameeteman, Pieter W., Coppen, Emma, Winder, Jessica Y., Moerland, Matthijs, Kan, Hermien, Grond, Jeroen, Webb, Andrew, Roos, Raymund A.C., Groeneveld, Geert Jan
Published in British journal of clinical pharmacology (01.05.2021)
Published in British journal of clinical pharmacology (01.05.2021)
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METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEURODEGENERATIVE DISEASE
WAKEFIELD, James, REDMON, Martin, ZARIWALA, Hatim A, ABBRUSCATO, Anthony
Year of Publication 02.05.2024
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Year of Publication 02.05.2024
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