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

Journal article

Open access

Peer reviewed

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

Orphanet journal of rare diseases, Vol.17(1), pp.1-336

02/09/2022

PMID: 36056411

Abstract

Barth syndrome

Elamipretide

Natural history control

TAZPOWER

url

https://doi.org/10.1186/s13023-022-02469-5View

Published (Version of record) Open

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Title: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome
Creators - without role: Brittany Hornby - Kennedy Krieger Institute
William Reid Thompson - Johns Hopkins Medicine
Mohammed Almuqbil - King Saud bin Abdulaziz University for Health Sciences
Ryan Manuel - Johns Hopkins Medicine
Anthony Abbruscato - Stealth BioTherapeutics
Jim Carr - Stealth BioTherapeutics
Hilary J. Vernon - Johns Hopkins Medicine
Publication Details: Orphanet journal of rare diseases, Vol.17(1), pp.1-336
Publisher: BioMed Central
Identifiers: 9920687408331
Academic Unit: King Abdulaziz University; King Saud Bin Abdulaziz University for Health Sciences
Language: English
Resource Type: Journal article