Search Results - "Guridi, Maitea"

The neuromuscular junction is a focal point of mTORC1 signaling in sarcopenia by Ham, Daniel J., Börsch, Anastasiya, Lin, Shuo, Thürkauf, Marco, Weihrauch, Martin, Reinhard, Judith R., Delezie, Julien, Battilana, Fabienne, Wang, Xueyong, Kaiser, Marco S., Guridi, Maitea, Sinnreich, Michael, Rich, Mark M., Mittal, Nitish, Tintignac, Lionel A., Handschin, Christoph, Zavolan, Mihaela, Rüegg, Markus A.

“…With human median lifespan extending into the 80s in many developed countries, the societal burden of age-related muscle loss (sarcopenia) is increasing…”
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Sustained Activation of mTORC1 in Skeletal Muscle Inhibits Constitutive and Starvation-Induced Autophagy and Causes a Severe, Late-Onset Myopathy by Castets, Perrine, Lin, Shuo, Rion, Nathalie, Di Fulvio, Sabrina, Romanino, Klaas, Guridi, Maitea, Frank, Stephan, Tintignac, Lionel A., Sinnreich, Michael, Rüegg, Markus A.

“…Autophagy is a catabolic process that ensures homeostatic cell clearance and is deregulated in a growing number of myopathological conditions. Although FoxO3…”
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mTORC1 and PKB/Akt control the muscle response to denervation by regulating autophagy and HDAC4 by Castets, Perrine, Rion, Nathalie, Théodore, Marine, Falcetta, Denis, Lin, Shuo, Reischl, Markus, Wild, Franziska, Guérard, Laurent, Eickhorst, Christopher, Brockhoff, Marielle, Guridi, Maitea, Ibebunjo, Chikwendu, Cruz, Joseph, Sinnreich, Michael, Rudolf, Rüdiger, Glass, David J., Rüegg, Markus A.

“…Loss of innervation of skeletal muscle is a determinant event in several muscle diseases. Although several effectors have been identified, the pathways…”
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Activation of mTORC1 in skeletal muscle regulates whole-body metabolism through FGF21 by Guridi, Maitea, Tintignac, Lionel A, Lin, Shuo, Kupr, Barbara, Castets, Perrine, Rüegg, Markus A

“…Skeletal muscle is the largest organ, comprising 40% of the total body lean mass, and affects whole-body metabolism in multiple ways. We investigated the…”
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Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy by Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca

“…Abstract Background In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment…”
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Differential response of skeletal muscles to mTORC1 signaling during atrophy and hypertrophy by Bentzinger, C Florian, Lin, Shuo, Romanino, Klaas, Castets, Perrine, Guridi, Maitea, Summermatter, Serge, Handschin, Christoph, Tintignac, Lionel A, Hall, Michael N, Rüegg, Markus A

“…Skeletal muscle mass is determined by the balance between protein synthesis and degradation. Mammalian target of rapamycin complex 1 (mTORC1) is a master…”
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O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD by O’Rourke, Erin, Proud, Crystal, Zaidman, Craig, Shieh, Perry B., McDonald, Craig, Day, John W., Mason, Stefanie, Guridi, Maitea, Han, Lixin, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Richardson, James, Malhotra, Jyoti, Singh, Teji, Rodino-Klapac, Louise, Mendell, Jerry

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Alterations to mTORC1 signaling in the skeletal muscle differentially affect whole-body metabolism by Guridi, Maitea, Kupr, Barbara, Romanino, Klaas, Lin, Shuo, Falcetta, Denis, Tintignac, Lionel, Rüegg, Markus A

“…The mammalian target of rapamycin complex 1 (mTORC1) is a central node in a network of signaling pathways controlling cell growth and survival. This…”
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Stride Velocity 95th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy by Rabbia, Michael, Guridi Ormazabal, Maitea, Staunton, Hannah, Veenstra, Klaas, Eggenspieler, Damien, Annoussamy, Mélanie, Servais, Laurent, Strijbos, Paul

“…Stride Velocity 95th Centile (SV95C) is the first wearable device-derived clinical outcome assessment (COA) to receive European Medicines Agency (EMA)…”
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Development of a Clinical Global Impression of Change measure for ambulant individuals with Duchenne muscular dystrophy by Staunton, Hannah, Trennery, Claire, Arbuckle, Rob, Guridi, Maitea, Zhuravleva, Elena, Furlong, Pat, Fischer, Ryan, Hall, Rebecca

“…In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often…”
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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study SRP‐9001‐103 (ENDEAVOR) by Zaidman, Craig M., Proud, Crystal M., McDonald, Craig M., Lehman, Kelly J., Goedeker, Natalie L., Mason, Stefanie, Murphy, Alexander P., Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle A., Potter, Rachael A., Rodino‐Klapac, Louise R., Mendell, Jerry R.

“…Objective Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4–5 years) with Duchenne muscular dystrophy. ENDEAVOR…”
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EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012) by Muntoni, Francesco, Mercuri, Eugenio, Schmidt, Ulrike Schara, Komaki, Hirofumi, Richardson, James, Singh, Tejdip, Guridi, Maitea, Mason, Stefanie, Murphy, Alex, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Mendell, Jerry

“…Abstract only…”
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One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) (S48.003) by Zaidman, Craig, Proud, Crystal, McDonald, Craig, Mason, Stefanie, Guridi, Maitea, Wang, Shufang, Reid, Carol, Darton, Eddie, Wandel, Christoph, Lewis, Sarah, Malhotra, Jyoti, Griffin, Danielle, Potter, Rachael, Rodino-Klapac, Louise, Mendell, Jerry

“…Abstract only…”
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Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD) (S48.006) by Proud, Crystal, Zaidman, Craig, Shieh, Perry, McDonald, Craig, Day, John, Mason, Stefanie, Guridi, Maitea, Han, Lixin, Yu, Lixi, Reid, Carol, Darton, Eddie, Wandel, Christoph, Richardson, James, Malhotra, Jyoti, Singh, Tejdip, Rodino-Klapac, Louise, Mendell, Jerry

“…Abstract only…”
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Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials by Servais, Laurent, Yen, Karl, Guridi, Maitea, Lukawy, Jacek, Vissière, David, Strijbos, Paul

“…In 2019, stride velocity 95th centile (SV95C) became the first wearable-derived digital clinical outcome assessment (COA) qualified by the European Medicines…”
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial by Mendell, Jerry R, Muntoni, Francesco, McDonald, Craig M, Mercuri, Eugenio M, Ciafaloni, Emma, Komaki, Hirofumi, Leon-Astudillo, Carmen, Nascimento, Andrés, Proud, Crystal, Schara-Schmidt, Ulrike, Veerapandiyan, Aravindhan, Zaidman, Craig M, Guridi, Maitea, Murphy, Alexander P, Reid, Carol, Wandel, Christoph, Asher, Damon R, Darton, Eddie, Mason, Stefanie, Potter, Rachael A, Singh, Teji, Zhang, Wenfei, Fontoura, Paulo, Elkins, Jacob S, Rodino-Klapac, Louise R

“…Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of…”
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Blocking the Interaction between Apolipoprotein E and Aβ Reduces Intraneuronal Accumulation of Aβ and Inhibits Synaptic Degeneration by Kuszczyk, Magdalena A, Sanchez, Sandrine, Pankiewicz, Joanna, Kim, Jungsu, Duszczyk, Malgorzata, Guridi, Maitea, Asuni, Ayodeji A, Sullivan, Patrick M, Holtzman, David M, Sadowski, Martin J

“…Accumulation of β-amyloid (Aβ) in the brain is a key event in Alzheimer disease pathogenesis. Apolipoprotein (Apo) E is a lipid carrier protein secreted by…”
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Antioxidant peroxiredoxin 6 protein rescues toxicity due to oxidative stress and cellular hypoxia in vitro, and attenuates prion-related pathology in vivo by Asuni, Ayodeji A., Guridi, Maitea, Sanchez, Sandrine, Sadowski, Martin J.

“…Protein misfolding, mitochondrial dysfunction and oxidative stress are common pathomechanisms that underlie neurodegenerative diseases. In prion disease,…”
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Modulation of amyloid precursor protein expression reduces β-amyloid deposition in a mouse model by Asuni, Ayodeji A., Guridi, Maitea, Pankiewicz, Joanna E., Sanchez, Sandrine, Sadowski, Martin J.

“…Objective Proteolytic cleavage of the amyloid precursor protein (APP) generates β‐amyloid (Aβ) peptides. Prolonged accumulation of Aβ in the brain underlies…”
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Home‐based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks by Contesse, Marielle G., Hodges, Jason, Staunton, Hannah, Lowes, Linda P., Elmankabadi, Bassem, Elder, Sonya J., Ormazabal, Maitea Guridi, Dalle Pazze, Laura, Leffler, Mindy G.

“…Background and Purpose Patients with Duchenne muscular dystrophy (DMD) change their movement patterns to compensate for muscle weakness. The Duchenne Video…”
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