Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials

Author:

Berry-Kravis Elizabeth1,Des Portes Vincent23,Hagerman Randi4,Jacquemont Sébastien56,Charles Perrine7,Visootsak Jeannie8,Brinkman Marc9,Rerat Karin10,Koumaras Barbara11,Zhu Liansheng12,Barth Gottfried Maria13,Jaecklin Thomas14,Apostol George14,von Raison Florian14

Affiliation:

1. Departments of Pediatrics, Neurological Sciences, and Biochemistry, Rush University Medical Center, Chicago, IL 60612, USA.

2. National Reference Center for Fragile X and Other X-Linked Intellectual Disabilities, CIC 1407 INSERM, Hospices Civils de Lyon, Université de Lyon, 69002 Lyon, France.

3. CNRS UMR 5304 (Laboratoire sur le Langage, le Cerveau et la Cognition), 69500 Bron, France.

4. Medical Investigation of Neurodevelopmental Disorders Institute and Department of Pediatrics, UC Davis Medical Center, Sacramento, CA 95817, USA.

5. Centre Hospitalier Universitaire Vaudois, CH-1011 Lausanne, Switzerland.

6. CHU Sainte-Justine Research Centre, Montreal, Quebec H3T 1C5, Canada.

7. Département de Génétique et Cytogénétique, Unité Fonctionnelle de Génétique Médicale, Assistance Publique–Hôpitaux de Paris, Groupe Hospitalier Pitié-Salpêtrière, 75013 Paris, France.

8. Department of Human Genetics, Emory University School of Medicine, Atlanta, GA 30322, USA.

9. Novartis Pharma GmbH, 90429 Nürnberg, Germany.

10. Novartis Pharmaceuticals SAS, 92500 Rueil-Malmaison, France.

11. Neurodegeneration Global Development, Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936–1080, USA.

12. Integrated Quantitative Science, Global Development, Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936–1080, USA.

13. Department of Child and Adolescent Psychiatry, University Hospital of Tübingen, 72076 Tübingen, Baden-Württemberg, Germany.

14. Neuroscience Development, Novartis Pharma AG, CH-4056 Basel, Switzerland.

Abstract

In contrast to previous studies, targeting the mGluR pathway in fragile X syndrome patients did not improve behavior independent of FMR1 methylation.

Funder

Novartis Pharma AG

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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