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Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial.
Thornton, Charles A; Moxley, Richard Thomas; Eichinger, Katy; Heatwole, Chad; Mignon, Laurence; Arnold, W David; Ashizawa, Tetsuo; Day, John W; Dent, Gersham; Tanner, Matthew K; Duong, Tina; Greene, Ericka P; Herbelin, Laura; Johnson, Nicholas E; King, Wendy; Kissel, John T; Leung, Doris G; Lott, Donovan J; Norris, Daniel A; Pucillo, Evan M; Schell, Wendy; Statland, Jeffrey M; Stinson, Nikia; Subramony, Sub H; Xia, Shuting; Bishop, Kathie M; Bennett, C Frank.
Afiliação
  • Thornton CA; Neurology, University of Rochester, Rochester, NY, USA.
  • Moxley RT; Neurology, University of Rochester, Rochester, NY, USA.
  • Eichinger K; Neurology, University of Rochester, Rochester, NY, USA.
  • Heatwole C; Center for Health and Technology, University of Rochester, Rochester, NY, USA.
  • Mignon L; Translational Medicine, Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Arnold WD; Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
  • Ashizawa T; Neuroscience Research Program, Houston Methodist Research Institute, Houston, TX, USA.
  • Day JW; Neuromuscular Medicine, Stanford University, Palo Alto, CA, USA.
  • Dent G; Neurodegeneration Development Unit, Biogen, Cambridge, MA, USA.
  • Tanner MK; Neurology, University of Rochester, Rochester, NY, USA.
  • Duong T; Neuromuscular Medicine, Stanford University, Palo Alto, CA, USA.
  • Greene EP; Neuromuscular Clinic, Houston Methodist Research Institute, Houston, TX, USA.
  • Herbelin L; Neurology, University of Kansas Medical Center, Kansas City, KS, USA.
  • Johnson NE; Neurology, University of Utah, Salt Lake City, UT, USA.
  • King W; Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
  • Kissel JT; Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA.
  • Leung DG; Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD, USA.
  • Lott DJ; Physical Therapy, University of Florida, Gainesville, FL, USA.
  • Norris DA; Pharmacokinetics and Clinical Pharmacology, Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Pucillo EM; Neurology, University of Utah, Salt Lake City, UT, USA.
  • Schell W; Neuromuscular Clinic, Houston Methodist Research Institute, Houston, TX, USA.
  • Statland JM; Neurology, University of Kansas Medical Center, Kansas City, KS, USA.
  • Stinson N; Center for Genetic Muscle Disorders, Kennedy Krieger Institute, Baltimore, MD, USA.
  • Subramony SH; Fixel Institute for Neurological Diseases, University of Florida, Gainesville, FL, USA.
  • Xia S; Biometrics, Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Bishop KM; Clinical Development, Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Bennett CF; Ionis Pharmaceuticals, Carlsbad, CA, USA. Electronic address: fbennett@ionisph.com.
Lancet Neurol ; 22(3): 218-228, 2023 03.
Article em En | MEDLINE | ID: mdl-36804094
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Distrofia Miotônica Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adult / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Distrofia Miotônica Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adult / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article