Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome

por Hornby, Brittany, Thompson, William Reid, Almuqbil, Mohammed, Manuel, Ryan, Abbruscato, Anthony, Carr, Jim, Vernon, Hilary J.
Publicado 2022

Safety, pharmacokinetics and pharmacodynamics of SBT‐020 in patients with early stage Huntington's disease, a 2‐part study

por van Diemen, Marcus P.J., Hart, Ellen P., Abbruscato, Anthony, Mead, Liz, van Beelen, Ilse, Bergheanu, Sandrin C., Hameeteman, Pieter W., Coppen, Emma, Winder, Jessica Y., Moerland, Matthijs, Kan, Hermien, van der Grond, Jeroen, Webb, Andrew, Roos, Raymund A.C., Groeneveld, Geert Jan
Publicado 2020