Patient Registries and Trial Readiness in Myotonic Dystrophy--TREAT-NMD/Marigold International Workshop Report.
Thompson
Rachel
R
TREAT-NMD, Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, NE1 3BZ, UK.
Schoser
Benedikt
B
Monckton
Darren G
DG
Blonsky
Karla
K
Lochmüller
Hanns
H
D002986:Clinical Trials as Topic; D019992:Databases as Topic; D006801:Humans; D007391:International Cooperation; D009223:Myotonic Dystrophy / Q000175:diagnosis / Q000235:genetics / Q000628:therapy; D012042:Registries; D012720:Severity of Illness Index; D016896:Treatment Outcome