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Pompe Disease: New Developments in an Old Lysosomal Storage Disorder
Meena NK, Raben N
Biomolecules, 2020, 10, 9
Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease
Meena NK, Ralston E, Raben N, et al.
Molecular therapy. Methods & clinical development, 2020, 18, p 199
Advancements in AAV-mediated Gene Therapy for Pompe Disease
Salabarria SM, Nair J, Clément N, et al.
Journal of Neuromuscular Diseases, 2019
Editorial for focused issue "Pompe disease: from basics to current and emerging therapies"
Puertollano R, Raben N
Annals of translational medicine, 2019, 7, 13, p 275
Pros and cons of different ways to address dysfunctional autophagy in Pompe disease
Lim JA, Meena NK, Raben N
Annals of translational medicine, 2019, 7, 13, p 279
Improved efficacy of a next-generation ERT in murine Pompe disease
Xu S, Lun Y, Frascella M, et al.
JCI insight, 2019, 4, 5
Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid alpha-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
Lim JA, Yi H, Gao F, et al.
Molecular therapy. Methods & clinical development, 2019, 12, p 233
Pompe Disease: From Basic Science to Therapy
Kohler L, Puertollano R, Raben N
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2018, 15, 4, p 928
Pompe disease: how to solve many problems with one solution
Puertollano R, Puertollano R, Raben N
Annals of translational medicine, 2018, 6, 5
Therapeutic Benefit of Autophagy Modulation in Pompe Disease
Lim JA, Sun B, Puertollano R, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018
Steps forward in Pompe disease. Abstracts of the 6th European Symposium. Berlin, Germany. November 23-24, 2012
Carlier RY, Mongini T, Vissing J, et al.
Congrès : 6th European Symposium : Steps forward in Pompe disease (23-24 November 2012; Berlin, Germany)
BMC musculoskeletal disorders, 2013, 14, suppl 2, 01-12
Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease
Spampanato C, Feeney E, Li L, et al.
EMBO Molecular Medicine, 2013, 5, 5, p. 691-706
Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy
Prater SN, Patel TT, Buckley AF, et al.
Orphanet journal of rare diseases, 2013, 8, 90, p. 1-12
Restoration of muscle functionality by genetic suppression of glycogen synthesis in a murine model of Pompe disease
Douillard-Guilloux G, Raben N, Takikita S, et al.
Human molecular genetics, 2010, 19, 4, p. 684-696
Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder-murine Pompe disease
Raben N, Schreiner C, Baum R, et al.
Autophagy, 2010, 6, 8, p. 1078-1089
Genetic suppression of autophagy dramatically enhances enzyme replacement therapy in murine Pompe disease (Steps forward in Pompe disease 3rd European Symposium)
Raben N, Baum R, Schreiner C, et al.
Congrès : Steps forward in Pompe disease 3rd European Symposium (20-21/11/2009; Munich (Allemagne))
Clinical Therapeutics, 2010, 32, Suppl. B, p. S62
Impaired organization and function of myofilaments in single muscle fibers from a mouse model of Pompe disease
Xu S, Galperin M, Melvin G, et al.
Journal of applied physiology, 2010, 108, 5, p. 1383-1388
Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease : implications for therapy
Raben N, Ralston E, Chien YH, et al.
Molecular genetics and metabolism, 2010, 101, 4, p. 324-331
When more is less. Excess and deficiency of autophagy coexist in skeletal muscle in Pompe disease
Raben N, Baum R, Schreiner C, et al.
Autophagy, 2009, 5, 1, p. 111-113
Autophagy in skeletal muscle : implications for Pompe disease
Shea L, Raben N
International journal of clinical pharmacology and therapeutics, 2009, 47, suppl 1, p. S42-S47
Murine muscle cell models for Pompe disease and their use in studying therapeutic approaches
Takikita S, Myerowitz R, Zaal K, et al.
Molecular genetics and metabolism, 2009, 96, 4, p. 208-217
Therapeutic approaches in glycogen storage disease type II/Pompe disease
Schoser B, Hill V, Raben N
Neurotherapeutics, 2008, 5, 4, p. 569-578
Dysferlin deficiency enhances monocyte phagocytosis. A model for the inflammatory onset of limb-girdle muscular dystrophy 2B
Nagaraju K, Rawat R, Veszelovszky E, et al.
American journal of pathology (The), 2008, 172, 3, p. 774-785
A new look at the pathogenesis of Pompe disease
Raben N, Plotz PH
Clinical Therapeutics, 2008, 30, Suppl C, p. S86-S87
A novel therapeutic approach for the glycogen storage disease type II
Douillard-Guilloux G, Batista L, Raben N, et al.
Congrès : Congrès international de myologie 2008 (International Congress of Myology 2008; 26-30 mai 2008; Marseille, France)
2008, p. 356
