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Expert Opinion on Orphan Drugs Volume 3, 2015 - Issue 9
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Review

Investigative and emerging treatments for Batten disease

Marta A TarczylukInstitute of Psychiatry, Psychology and Neuroscience, Pediatric Storage Disorders Laboratory, King’s College London, James Black Centre, Department of Basic and Clinical Neuroscience, 125 Coldharbour Lane, London, SE5 9NU, UK
, PhD (Postdoctoral Research Worker) &
Jonathan D CooperInstitute of Psychiatry, Psychology and Neuroscience, Pediatric Storage Disorders Laboratory, King’s College London, James Black Centre, Department of Basic and Clinical Neuroscience, 125 Coldharbour Lane, London, SE5 9NU, UK;Maurice Wohl Clinical Neuroscience Institute, Institute of Psychiatry, Psychology and Neuroscience, Pediatric Storage Disorders Laboratory, Department of Basic and Clinical Neuroscience, King’s College London, 5 Cutcombe Road, London, SE5 9RX, [email protected]
, PhD (Professor of Experimental Neuropathology)
Pages 1031-1045 | Published online: 27 Jul 2015

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Jonathan B. Rosenberg, Alvin Chen, Stephen M. Kaminsky, Ronald G. Crystal & Dolan Sondhi. (2019) Advances in the treatment of neuronal ceroid lipofuscinosis. Expert Opinion on Orphan Drugs 7:11, pages 473-500.
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Articles from other publishers (3)

Nicola Specchio, Alessandro Ferretti, Marina Trivisano, Nicola Pietrafusa, Chiara Pepi, Costanza Calabrese, Susanna Livadiotti, Alessandra Simonetti, Paolo Rossi, Paolo Curatolo & Federico Vigevano. (2020) Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy. Drugs 81:1, pages 101-123.
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Dolan Sondhi, Stephen M. KaminskyNeil R. HackettOdelya E. PagovichJonathan B. RosenbergBishnu P. DeAlvin ChenBenjamin Van de GraafJason G. MezeyGrace W. MammenDenesy MancenidoFang XuBarry KosofskyKaleb Yohay, Stefan Worgall, Robert J. Kaner, Mark Souwedaine, Bruce M. GreenwaldMichael Kaplitt, Jonathan P. Dyke, Douglas J. Ballon, Linda A. Heier, Szilard Kiss & Ronald G. Crystal. (2020) Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2 . Science Translational Medicine 12:572.
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Marta A. Tarczyluk-Wells, Christoph Salzlechner, Allison R. Najafi, Ming J. Lim, David Smith, Frances M. Platt, Brenda P. Williams & Jonathan D. Cooper. (2019) Combined Anti-inflammatory and Neuroprotective Treatments Have the Potential to Impact Disease Phenotypes in Cln3−/− Mice. Frontiers in Neurology 10.
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