Global FKRP Registry: observations in more than 300 patients with Limb Girdle Muscular Dystrophy R9.
Lindsay B MurphyOlivia Schreiber-KatzKaren RaffertyAgata RobertsonAna TopfTracey A WillisMarcel HeidemannSimone ThieleLaurence BindoffJean-Pierre LaurentHanns LochmüllerKatherine MathewsClaudia MitchellJohn Herbert StevensonJohn VissingLacey WoodsMaggie C WalterVolker StraubPublished in: Annals of clinical and translational neurology (2020)
The Global FKRP Registry enables the collection of patient natural history data, which informs academics, healthcare professionals and industry. It represents a trial-ready cohort of individuals and is centrally placed to facilitate recruitment to clinical studies.