Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease by Paul Wuh‐Liang Hwu, Karl Kiening, Irina Anselm, David R Compton, Takeshi Nakajima, Thomas Opladen, Phillip L Pearl, Agathe Roubertie, Thomas Roujeau, Shin‐ichi Muramatsu

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Patient selection considerations for AADC deficiency gene therapy by Agathe Roubertie, Irina Anselm, Bruria Ben‐Zeev, Wuh‐Liang Hwu, Ashutosh Kumar, Berrin Monteleone, Shin‐ichi Muramatsu, Vincenzo Leuzzi, Salvador Ibáñez, Scellig Stone, Phillip L. Pearl

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Novel Mutation at Cys225 in GNAO1‐Associated Developmental and Epileptic Encephalopathies: Clinical, Molecular, and Pharmacological Profiling of Case Studies by Yonika A. Larasati, Gonzalo P. Solis, Alexey Koval, Marie‐Céline François‐Heude, Julie Piarroux, Agathe Roubertie, Ruihan Yang, Ying Zhang, Dezhi Cao, Christian M. Korff, Vladimir L. Katanaev

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Neurofibromatosis-Noonan syndrome: a prospective monocentric study of 26 patients and literature review by Didier Bessis, Dominique Vidaud, Pierre Meyer, Laurence Pacot, de La Villeon G, Adeline Alice Bonnard, Yline Capri, Christine Coubes, Fanchon Herman, Didier Lacombe, Nicolas Molinari, Laura Poujade, Agathe Roubertie, Julien Van Gils, Alain Verloes, David Geneviève, Hélène Cavé, Marjolaine Willems

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Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severi... by Eugen Mengel, Marc C. Patterson, Rosalia M. Da Riol, Mireia Del Toro, Federica Deodato, Matthias Gautschi, Stephanie Grunewald, Sabine Weller Grønborg, Paul Harmatz, Julia B. Hennermann, Bénédicte Héron, Esther M. Maier, Agathe Roubertie, Saikat Santra, Anna Tylki-Szymanska, Lisa LaGorio, Elizabeth Berry-Kravis, Forbes D. Porter, Beth Solomon, Louise Himmelstrup, Travis Mickle, Sven Guenther, Christine í Dali

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Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind c... by Una Martin, Timothy Barrett, Susan Mollan, Karen E Morrison, Ruth Evans, Isabelle Meunier, Rajat Gupta, Andrew Peet, Martin Wilson, Sulev Koks, Wojciech Młynarski, Patrick Yu-Wai-Man, Agathe Roubertie, Darren Barton, Dewi Astuti, Renuka P Dias, Lucinda Billingham, Kun Hu, Malgorzata Zatyka, Archana Kulkarni, Benjamin Wright, Kristian Brock, Victoria Homer, Rebecca Malpass, Heather Rose, Christophe Orssaud, Zsuzsanna Nagy, Tracy Lynch, Sabrina Cronier, Amandip Malhi, Pooja Takhar, Amy Lamb, Gema Esteban-Bueno

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