Abstract Reference and Poster Numbers

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1. Observational study in adults with spinal muscular atrophy treated with Risdiplam.  Rodrigue X, Henley K.  
2. A Five-Year Update on a Real-World Study of Canadian Adults with Spinal Muscular Atrophy Type 2 and 3 Treated with Nusinersen. Rodrigue X, Charron C.
3. Positive predictive value of myositis antibody line blot testing in patients with suspected idiopathic inflammatory myopathy. Chang Y, Yang L, Budhram A.
4. NADMED: Targeted REDOX profiling for clinical and research use.  Äänismaa R, Jansson S, Buzkova J, Euro L, Suomalainen A.
5. Rewiring myogenic cell identity: Comparative analysis of PAX7 and PAX7-FOXO1 in Rhabdomyosarcoma and muscle progenitors. Ghavidel P and Sincennes M-C.
6. Thoracic Electric Impedance Tomography Detects Lung Volume Changes in Amyotrophic Lateral Sclerosis. Hansen G, Shaw A, Bolt K, Verity R, Nataraj RT, Schellenberg KL.
7. Best practice recommendations for the clinical care of spinal bulbar muscular atrophy.  Schellenberg KL, Caspar-Bell G, Ellis C, Johnston W, King A, King M, Korngut L, Kushneriuk B, Lavoie AJ, McGonigle R, Newton J, O’Connell C, Shoesmith C, Suchowersky O, Warman-Chardon J, Wunder S, Pfeffer G.
8. Longitudinal analysis of glymphatic function in amyotrophic lateral sclerosis and primary lateral sclerosis. Sharkey RJ, Cortese F, Goodyear BG, Korngut LW, Jacob SM, Sharkey KA, Kalra S, Nguyen MD, Frayne R, Pfeffer G. Presented by: Soule, T.
9. Higher than expected incident cases of spinal bulbar muscular atrophy in western Canada. Lamont R, King M, King A, Schellenberg K, Pfeffer G. Presented by: Soule, T.
10. The oral microbiome in ALS shows differentially abundant organisms in limb versus bulbar onset disease: a binational study. Jacob SM, Son B, Bagheri S, Lee S, Leckie J, Chohan B, Belway C, Mascarenhas J, Mobach T, Korngut LW, Sharkey KA, Park J, Nguyen MD, Kim SH, Pfeffer G. Presented by: Soule, T.
11. Results of VBP15-006: A Phase 2, Open-Label, Multiple Dose Study of Vamorolone in Boys With DMD Aged 2 to <4 and 7 to <18 Years. Mah JK, Gonorazky HD, Lochmüller H, Aleman A, Yaworski A, Oskoui M, Sbrocchi AM, de Vera A, Dutreix C, Gresko E, Linden A, Berglund A, Charef P, Hoffman E, Damsker J, Selby K.
12. Modeling Fetal Acetylcholine Receptor Inactivation Syndrome (FARIS) Using Patient-Derived iPSCs to Elucidate Pathogenesis and Identify Therapeutic Targets. Ouhaddi Y, Orfi Z, Campeau PM, Nguyen CE, Dumont NA.
13. Sustained functional improvement with DYNE-251 in males with DMD mutations amenable to exon 51 skipping enrolled in the Phase 1/2 DELIVER trial. McMillan H, Campbell C, Deconinck N, De Waele L, Flanigan K, Lorentzos M, Phan H, Shieh P, Ray S, Wang D, Dugar A, Naylor ML, Kerr D, Guglieri M, on behalf of the DELIVER study investigators.  
14. Novel prodrug improves locomotor disfunction in gne deficient zebrafish. Lau J, Derkson A, O’Connor E, Morewood J, Spendiff S, Pertusati F, Lochmüller H.
15. Galactose treatment rescues neuromuscular junction transmission in glutamine-fructose-6-phosphate transaminase 1 (Gfpt1) deficient mice. Holland SH, Carmona-Martinez R, O’Neil D, Ho K, O’Connor K, Azuma Y, Roos A, Spendiff S, Lochmüller H.
16. Unraveling the Function of MLIP in Myogenic Cells: Toward Understanding Its Role in Rare Myopathy. Bachir AY, Sleno L, Tétreault M, Sincennes M-C.   
17. Correcting mutations responsible for LGMD R2/2B (dysferlinopathy) with Prime editing.  Bouchard C, Jiang J, Rousseau J, Tremblay JP.
18. Muscle-specific allelic imbalance of IARS1 unmasks a novel compound heterozygous mechanism in congenital myopathy. Rihoux A, Gonthier-Cummings C, Hauteclocque J, Gagné A, Hamilton LK, Karamchandani J, Labarre A, Parker A, Brais B, Tétreault M. 
19. Systemic CRISPR/SaCas9-mediated exon skipping achieves long-term benefit in canine Duchenne muscular dystrophy.   Maruyama R, Moriyama H, Minegishi K, Wilton-Clark H, Ashida Y, Kuraoka M, Aoki Y, Yokota T.   
20. Maternal transfer of disease modifying therapies for spinal muscular atrophy: a novel, minimally invasive approach to deliver SMN therapy prenatally. Sutton ER, Beauvais A, De Repentigny Y, Yaworski R, McMillan H, Schneider B, Mattar P, Kothary R.
21. Creating an open-access SOP resource for neuromuscular disease researchers. Sutton E, Rihoux A, Yaworski R, Lee J, Cusmano A, Atieh S, Triolo M, Leduc-Gaudet J-P, Perry CGR, Patten K, Kothary R, Chang NC.
22. A Canadian Perspective on the Diagnosis and Management of Mixed Phenotype Hereditary Transthyretin Amyloidosis1. Mezei M, Bril V, Ducharme A, Fine N, Matte G, Delgado D.
23. Autosomal dominant rhabdomyolysis is associated with a missense variant in the ATP2A2 reducing SERCA2 calcium pump function in skeletal muscle. Idoux R, Malaichamy S, Polavarapu K, Thompson R, Spendiff S, Freeman E, O’Neil D, Carmona-Martinez R, Roos A, Barić I, Lochmüller H.
24. Developing and optimizing a structured therapeutic platform for individualized gene editing therapies in DMD: Integrating therapeutic innovation and ethical implementation. Perillat L, Rok M, Nolasco G, Kozdas B, Ivakine E, Cohn R.    
25. Systematic Reanalysis of NGS Data in 103 NMD Families Enhances Diagnostic Yield, Reveals Deep Intronic Variants, and Identifies ATP2A2 as a Novel Neuromuscular Disease Gene.  Malaichamy S, Polavarapu K, Thompson R, Idoux R, Spendiff S, Karcagi V, Herczegfalvi A, Almomen M, Vengalil S, Nashi S, Laurie S, Matalonga L, Beltran S, Voermans N, Roos A, Barić I, Nalini A, Lochmüller H.
26. The Impact of Premature Cellular Senescence in Muscle Regeneration in Myotonic Dystrophy type 1. Kim T, Koike T, Conte T, Molina T, Mokhtari I, Duchesne E, Dumont N.
27. Impact of Trained Immunity on Macrophage and Muscle Stem Cell Function in Dystrophic (mdx) Mice. Deprez A, Bhattarai S, Liang F, Gusev E, Petrof BJ.
28. Inhibition of translation initiation factor eIF4A in dystrophic muscle stem cells drives cell fate towards differentiation. Filippelli RL, Robertson R, Liu Y, Granet JA, Cusmano AA, Gallouzi IE, Pelletier J, Chang NC.   
29. Exploring cardiac and skeletal muscle dysfunction in Myotonic Dystrophy Type 1 (DM1) with patient-derived iPSCs. Part CE, Geertsma H, Gutierrez L, Baird S, MacKenzie A, Spendiff S, Lochmüller H.
30. Understanding myotonic dystrophy type 1 using iPSC-derived neurons. Geertsma H, Part C, O’Connor K, Baird S, MacKenzie A, Spendiff S, Lochmuller H.
31. A novel homozygous variant in AHCY causes a rare muscular dystrophy: a new case. Triassi V, Ellezam B, Jizi K, Jacquemont S, Nguyen CE, Tetreault M.
32. TWEAK Administration Improves Muscle Function in D2.mdx mice. Lala-Tabbert N,  Whitney R, Robert N, Ratsun D, Humphrey A, Earl N, Korneluk RG.    
33. EV mediated delivery of Wnt7a as a therapy for muscle wasting diseases.    Javandoost E, Datzkiw D, Gurriaran-Rodriguez U, Rudnicki, MA,    
34. DG9-PMO Enhances Cardiac and Skeletal Muscle Delivery for Exon 44 Skipping Therapy in Duchenne Muscular Dystrophy. Shah MNA, Wilton-Clark H, Haque F, Powell B, Sutanto LE, Maradiya R, Zhabyeyev P, Roshmi RR, Anwar S, Aslesh T, Lim KRQ, Maruyama R, Bigot A, Young CS, Bittner S, Spencer MJ, Moulton HM, Oudit GY, Yokota T.
35. A Common Thread? Multi-Omics Uncover Denervation in Diverse Myopathies. Soule TGB, Pontifex CS, Rosin N, Pun A, Wuerch E, Ho Chu T, Chohan S, Jacob S, Dabaja M, Dufour A, Yong W, Biernaskie J, Pfeffer G.
36. A novel therapeutic effect of lauric acid: preventing loss of ambulation in severe mouse models of muscular dystrophy. Sun Z, White Z, Kang Y, Lyu Y, Bernatchez P.
37. Chronic loss of the mitochondrial fusion protein OPA1 is detrimental to muscle stem cell maintenance and activity. Sommers O, Triolo M, Wade S, Dort J, Khacho M.
38. Transition to home infusions with a low risk of infusion-associated reactions and functional stability: real-world outcomes from the UK Early Access to Medicines Scheme registry for cipaglucosidase alfa plus miglustat in late-onset Pompe disease. Roberts M, McPherson A, Jain V, Moffat E, Clarke S, Sitaraman Das S, Zhang J.
39. Senotherapeutics and Exercise-Based Strategies to Restore Defective Muscle Stem Cells in Myotonic Dystrophy Type 1. Koike T, Kim T, Mokhtari I, Conte T, Duchesne E, Dumont N.    
40. The Impact of DEPP1 Overexpression on Skeletal Muscle Function. Delisle P, Hevey A, Capobianco A, Pouliot C, Leduc-Gaudet JP.  
41. Identification and Characterization of Serglycin as a Potential Biomarker in GNE Myopathy. Derksen A, Spendiff S, Pogoryelova O, Hentschel A, Dobelmann V, Roos A, Lochmüller H.
42. Functional validation of congenital myasthenic syndrome candidate genes: A focus on ETV5 and ATP8A2. Palacek K, Lau J, Meyer S, Sivasankar M, Aksel-Kilicarslan O, Spendiff S, Lochmüller H.
43. Music Intervention for Brain-Heart Disease in Pediatric Myotonic Dystrophy Type 1 (DM1): Interim results on feasibility and tolerability. Eisenkölbl A, Burgess T, Howley H, Parkes E, Comeau G, Berard JA, Walker LAS, Woollam A, Yaworski A, McMillan H, Lochmüller H.
44. Assessing 2-point DIXON and 6-point PDFF fat fraction using full body muscle MRI analysis in patients with oculopharyngeal muscular dystrophy (OPMD). Esttaifo F, Khan S, Meier-Ross K, Melkus G, Sampaio ML, Smith IC, Warman-Chardon J.
45. A molecular switch: permeability transition pore dynamics regulates mitochondrial membrane potential and quiescence in muscle stem cells. Cairns G, Gourlay M, Larionov N, Burelle Y.     
46. Phase 1b study of the safety, tolerability, pharmacokinetics, immunogenicity, and efficacy of ARGX-119 in participants with DOK7 congenital myasthenic syndromes. Lochmuller H, Maggi L, Kuntz NL, Burden SJ, Van Bragt T, Dincq S, Talloen W, Lim J, Michiels H, Patel S, Orogun L, Raza S, Vanhauwaert R, Shilling R, Palace J.
47. Lipid profiling uncovers peroxisomal roles in metabolic reprogramming and muscle stem cell fate. Cairns G, Ly HM, Larionov N, Burelle Y.
48. Assessing suitability of different subgroups for interventional trials in Duchenne muscular dystrophy. Guite K, Hoffman EP, Guglieri M, Clemens PR, on behalf of VBP investigators, Dang UJ.
49. French-Canadian new founder mutation on DTNA gene associated with mild muscular dystrophy. Grimard O, Nguyen C-TE, Campeau P, Thuriot F, Lévesque S, Soucy J-F, Hamdan F, Gauthier JM. 
50. 48-week data from the Phase 2 open-label FORWARD-53 study of WVE-N531 in boys with Duchenne muscular dystrophy amenable to exon 53 skipping. Tai L-J, Servais L, Bader M, AlQurashi M, Goel V, Ghosh A, Ghosh D, Haegele JA, Hart A, Longo K, Narayanan P, Paulson D, Rheinhardt J, Singh K, Strahs A, Wagner E, Yang G, Vargeese C, Malhi S, Ingelsson E.
51. The Role of Genetic Modifiers in Drug Response Safety in Duchenne Muscular Dystrophy. Ahmadiharchegani F, Hoffman EP, Sabbatini D, Pegoraro E, Bello L, Guglieri M, Clemens P, on behalf of VBP investigators, Dang UJ.
52. Proximal Femoral Screw Hemiepiphysiodesis in Spinal Muscular Atrophy: A Case Report in the Gene Therapy Era. Nauleau-Javaudin A, Rioux-Trottier E, Fiscaletti M, Martineau-Beaulieu E, Pilon-Cadieux C, Nguyen C-TE.
53. Skeletal Muscle Transcriptomic Comparison Between Men and Women Individuals with Myotonic Dystrophy Type 1. Dupèbe W, Leduc-Gaudet JP.
54. Interpreting limb girdle muscular dystrophy R1 mutations in the calpain-3 structure. Bell M, Ye Q, Serrão V, Davies P.
55. Investigating a potential muscle-driven mechanism of neuromuscular denervation in ALS. Boyer M, Berthod F, Pozzi S.
56. Risdiplam clinical trial outcomes in individuals with spinal muscular atrophy (SMA) and four SMN2 copies. Toy F, Bertini E, Chiriboga CA, Mercuri E, Dickendesher T, Li Y, Martin C, Palfreeman L, Tam S, Yeung WY, Wilson M.
57. Sunfish Parts 1 and 2: 5-Year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy. Nascimento A, Day JW, Deconinck N, Mazzone ES, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Sully K, Kuthiala M, Gorni K, Martin C, Yeung WY, Scalco RS, Mercuri E, on behalf of the SUNFISH Study Group.
58. Muscle and bone growth in the lower legs of typically developing children and children with cerebral palsy: a longitudinal imaging study. Bolsterlee B, Chow BVY, Davies S, Morgan C, Rae CD, Warton DI, Novak I, Lancaster A, Popovic GC, Seydi M, Rizzo CY, Ball IK,Robert D. Herbert RD.  
59. A comprehensive and queryable database with thousands of findings on biomarkers from >20 serum and tissue datasets for DMD. Dang UJ, Tu W, Tobin R, Abdelrazeq L, Guite K, Szigyarto CAK, Tsonaka R, Degan C, van der Burgt Y, Diaz Manera J, Guglieri M, Spitali P, Hathout Y.
60. Differential Proteomic Profiling of Cerebrum and Myelin in the mdx Mouse Models of Duchenne Muscular Dystrophy. Alfarra A, Lyons C, Rijal A, Thangarajh M, Dang UJ.
61. Performance of the 2017 EULAR/ACR Classification Criteria in Indian Patients with Idiopathic Inflammatory Myopathies. Jassal B, Dhall A, Vishnu VY, Bhadu D, Suri V, Sharma MC.
62. Contributions of protein-S-glutathionylation to skeletal muscle differentiation.  Gardner E, Khacho M.
63. Epigenetic Role of Six1 in Muscle Stem Cell Activation. Acar K, Caceres D, Blais A.
64. Hydrogen sulfide rescues a common redox-dependent mechanism of eccentric contraction-induced force loss in Dmd(mdx) and Actb knockout mice. Fallon KS, Southern WM, Lindsay A, Lowe DA, Ervasti JM
65. Multispectral optoacoustic tomography as non-invasive biomarker for diagnosis and monitoring of neuromuscular diseases. Anderson ETE, Eisenkölbl A, Aksel Kilicarslan O, Burgess T, Carmona R, Tugirimana PL, Meyer S, Thornhill R, Yaworski A, McMillan H, Lochmüller H.
66. A case of amyloid myopathy as the initial manifestation of immunoglobulin light chain amyloidosis. Adler P, Rossiter J, Ren K, Melanson M.
67. A preclinical rationale for combining SMAC mimetics with radiation therapy to reprogram cold rhabdomyosarcoma tumours. Robert N, Di Tiero A, Humphrey A, Ratsun D, Lala-Tabbert N, De Lisio M, Korneluk RG.
68. Muscle-specific extracellular vesicles: a novel biomarker for spinal muscular atrophy. Duguez S, McCallion E, Duc P, Moisan A, Schneider BL, Rage F, Bowerman M.      
69. Engineering PBMC-derived skeletal muscle organoids as a model for Duchenne muscular dystrophy (DMD). Flynn-Rizk R, Egiian M, Hoffman L.
70. Functional characterization of a dystrophin-null mouse model, DMD-Null, for studying Duchenne muscular dystrophy. Wilton-Clark H, Shah MNA, Leckie J, Al-Agbhari A, Hernandez S, Zhabyev P, Maruyama R, Oudit G, Yokota T.
71. Population-based estimates of the direct and indirect costs of living with oculopharyngeal muscular dystrophy (OPMD) in Canada. Smith IC, Grant A, Lessard LER, Osman H, Lochmuller H, McMillan H, Pfeffer G, Korngut L, Gagnon C, Lintern S, Selby K, Thavorn K, Warman-Chardon J.
72. Early loss of intramuscular motor axons limits capacity for recovery following Smn up-regulating therapy in a mouse model of spinal muscular atrophy. Comley LH, Partlova I, Nyasa C, Pyrgioti M, Murray LM.
73. Autophagy maintains the regenerative capacity of muscle stem cells. Cusmano AA, Granet JA, VanGenderen CA, Robertson R, Chang NC.
74. Influence of age, CTG repeat, phenotype and training on the Splicing Index in DM1 patients. Légaré C, Planco L, Davey EE, Lennon C, Shaughnessy S, Girard-Côté L, Roussel MP, Cleary JD, Berglund JA, Duchesne E.
75. MOXIe clinical trial overview of Omaveloxolone for patients with Friedreich Ataxia. Escamilla C, Lubkov V, Jaramillo R. Presented by: Leyderman, K.
76. The MOXIe Trial of Omaveloxolone in Friedreich Ataxia: Exploring the Transient Nature of Treatment-Emergent Adverse Events. Lynch D, Boesch S, Delatycki M, Giunti P, Goldsberrya A, Hoyle C, Mathews K, Khana S, Meyera C, Muraia M, Nachbauer W, Perlman S, Subramony S, Zesiewicz T. Presented by: Leyderman, K.
77. Managing aminotransferase elevations in patients with friedreich ataxia treated with Omaveloxolone: expert opinion on use considerations. Lynch D, Anheim M, Boesch S, Lewis J, Perlman S. Presented by: Depres-Tremblay, G.
78. Strength training improves mitochondrial respiration, H2O2 emission and histological muscle integrity in women with Myotonic Dystrophy Type 1.  Marcangeli V, Girard-Côté L, Di Leo V, Roussel MP, Lawless C, Charest O, Argaw A, Dulac M, Hajj-Boutros G, Morais JA, Vincent A, Gouspillou G, Leduc-Gaudet J-P, Duchesne E. 
79. Investigating the role of aquaporin 1 and 4 in skeletal muscle function and pathology in Duchenne muscular dystrophy. Leckie J, Aslesh T, Shah A, Zia A, Standell T, Yokota T.
80. E3 Ligase Parkin mediates muscle stem cell lineage progression. Gourlay M, Abbasi M, Wang A, Cairns G, Burelle Y.
81. Investigating a miniaturized nebulin for nebulin-related nemaline myopathy. Al-Mudarris T, Fabian L, Bogaards SJP, Viththiyapaskaran S, Karimi E, Farman GP, Sabha N, Granzier HL, Ottenheijm CAC, Dowling, JJ.    
82. Tracking early oxygen dysregulation in DMD rat models: toward optical monitoring of disease progression. Egiian M, Eskandari R, Tyler S, Morrison L, Diop M, Hoffman L.
83. Identification of early symptomatic gene expression changes in motor neurons in a large animal model of motor neuron disease. Baird M, Fox A, McGovern V, Pastore B, Rocco M, Tanque A, Ceyhan K, Sran S, Acosta Martinez S, Mattox L, Lordi N, Joseph M, Bedrosian T, Deardorff A, Youngblood B, Samaranch L, Kolb S.
84. Presence of symptoms and signs of neuropathy in a cohort of patients with SCA27B. Iruzubieta P, Pellerin D, Ashton C, Villa F, Renaud M, Dicaire MJ, Danzi M, Aldecoa M, Mathieu J, Massie R, Chalk C, Lafontaine AL, Evoy F, Rioux MF, Brisson JD, Boycott K, Houlden H, Synofzik M, La Piana R, Zuchner S, Duquette A, Brais B.
85. Investigating the impact of muscle stem cell-specific mitochondrial dysfunction on inflammation during muscle regeneration. Tomsine RA, Larionov N, Milliken M, Sandhu J, Khacho M.
86. Limb-Girdle Muscular Dystrophies in a Single-Center Adult Population: Clinical Spectrum and Genetic Findings. Aldecoa M, Iruzubieta P, Brais B, O'Ferrall E.
87. Telemedicine and remote monitoring in neuromuscular disease care: Effectiveness and challenges in follow-up care and multidisciplinary coordination. Gabr M.
88. Train(e)d: Training Platform for Building National Clinical Outcome Assessment Capacity in Neuromuscular Disease. Masnata M, Adamji Z, Osman H, Beattie E, O’Connell C, Gagnon C, Warman-Chardon J.
89. Experience as evidence: mapping the patient journey of Friedreich ataxia in Canada. Adamji Z, Osman H, Lintern S.
90. Characterizing spinal cord pathology in the cuprizone autoimmune encephalomyelitis model: evaluating its potential as a comprehensive model for multiple sclerosis research. Al-Aarg M, Alves de Almeida MM, De Repentigny Y, Kothary R. 
91. A dosage study for aav9-mediated gene therapy and the development of combinatorial therapy for the treatment of spinal muscular atrophy. Yaworski R, Beauvais A, Sutton E, Lorson C, Schneider B, Kothary R.
92. ClC-1 chloride channel inhibitor NMD712 improves motor function in mouse models of congenital myasthenic syndromes Ho K, Carmona-Martinez R, Skov M, Spendiff S, & Lochmüller H.
93. Regulation of mitophagy by the NIX pathway during MuSC state transition. Dixon M, Racine J, Larionov N, Cairns G, Khacho M, Burelle Y.
94. Overview of the Canadian Neuromuscular Disease Registry (CNDR): Applying Real-World Evidence to Improve Disease Research and Outcomes in Canada. Langevin LM, Crone M, Hodgkinson V, Jewett G, Lochmüller H, McMillan H, Korngut L, on behalf of the CNDR Investigator Network.
95. Development of an in-silico workflow to characterize the spliceogenicity and protein effect of splice-altering variants. Zeldin J, Polavarapu K, Thompson R, Lochmüller H.  
96. The Glucocorticoid Receptor Regulates Nuclear Positioning During Skeletal Muscle Differentiation. Alharbi S, Wiper-Bergeron N. 
97. Patient MTRFR iPSC-derived neurons and muscle exhibit cell specific phenotypes and mitochondrial dysfunction. O’Connor K, Zarate-Mendez M, Spendiff S, Horvath R, Lochmüller H.
98. Recessive titinopathies: Clinical and molecular insights from seven patients with biallelic TTN variants. Aksel Kilicarslan O, Polavarapu K, Gangfuß A, Kölbel H, Thompson R, Peric S, Pauper M, Roos A, Lochmüller H.
99. What are the Costs of Living with FacioScapuloHumeral Dystrophy? The Socioeconomic Burden of FSHD. Lessard LER, Grant A, Smith IC, Osman H, Lochmüller Ha, McMillan H, Pfeffer G, Korngut L, Gagnon C, Lintern S, Selby K, Ari B, Thavorn K, Warman-Chardon J.
100. Dystrophic satellite cells undergo mitotic catastrophe and senescence. Pan W, Rudnicki M.
101. C9orf72 loss of function leads to cerebellar degeneration in the zebrafish ALS model. Singh J, Lescouzères L, Patten K.
102. Characterizing the role of the RNA-binding protein HuR in regulating the structure of neuromuscular junctions in Duchenne muscular dystrophy. Bhaura G, Gobin J, Ravel-Chapuis A, Jasmin B.
103. SAT-3247: An oral small molecule inhibitor targeting AAK1, a critical effector of skeletal muscle regeneration. Smid J, Rayagiri S, Kodippili K, Mitchell R, Jaquith J, Lambert P, Rudnicki M.
104. Identifying cancer cachexia humoral factors affecting muscle stem cell regeneration capacity. LoFaro M, Hall D, Rudnicki M.  
105. The comparative efficacy and safety of risdiplam versus high-dose nusinersen in children with Type 1 SMA. Kokaliaris C, Hawkins N, Mahajan A, Scott DA, Sideris CE.   Non-author local presenter: Inskip JA.
106. Deciphering the mechanisms involved in the AMPK-induced therapeutic benefits in Myotonic Dystrophy type 1 skeletal muscle. Fahmi C, Lessard L, Mounier R, Ravel-Chapuis A, Jasmin BJ.
107. Characterization of Bitter Melon Natural Compounds that Activate AMPK Signaling As Novel Therapeutics For Myotonic Dystrophy Type 1 (DM1). Atieh S, Liu J, Ye Y, Tang C, Ravel-Chapuis A, Jasmin BJ.
108. Characterization of a novel muscle stem cell subpopulation with low proliferative and metabolic profiles.   Lin T, Guilhot C, Ashekyan O, Lin AYT, Rudnicki MA.    
109. Therapeutic modulation of the calpastatin/calpain system restores neuromuscular integrity and function in C9orf72 ALS. Lescouzères L, Butti Z, Chaineau M, Young D, You Z, Nicouleau M, Chen CX-Q, Haghi G, Aprahamian N, Zaouter C, Dufour A, Durcan TM, Patten K.
110. Longitudinally tracking disease progression and correlation in oculopharyngeal muscular dystrophy patients using whole-body quantitative MRI. Satarkar T, Smith IC, Khan S, Esttaifo F, Meier-Ross K, Melkus G, Sampaio M, Warman-Chardon J.
111. Polarity dysregulation in dystrophin-deficient satellite cells. Catenacci M, Rudnicki MA.  
112. A muscular dystrophy associated with bi-allelic LEMD2 variants: Expanding the genotype of nuclear envelopathies. Pauper M, Kölbel H, Karakesisoglou I, Schänzer A, Böhm J, Thompson R, Kohlschmidt N, Ringel B, Bonne G, Neuhoff K, Gangfuß A, Aksel Kilicarslan O, Beltran Agullo S, Hentschel A, Schara-Schmidt U, Lochmüller H, Polavarapu K, Roos A.  
113. Factors that influence adherence to home-based rehabilitation in children and youth with neuromuscular disorders: a scoping review. Grangé A, Boulais J, Levac D.
114. Diagnostic Delay Leading to Inpatient ALS Diagnoses: A Case Series. Donaghy R, Seth A, Zhang l, Harris G, Sufit R, Kalb R, Ajroud-Driss S.    
115. Mitochondrial redox signaling regulates muscle stem cell function through glutathione-s-transferase pi1. Triolo M, Faraji D, Wade S, Larionov N, Khacho M.
116. Dystrophic muscle phenotypes can be horizontally transferred via fecal microbiome transplantations.   Butcher J, Gosse JT, Ravel-Chapuis A, Jasmin BJ, Stintzi A.  
117. Retrospective assessment of feeding and nutrition after 2 years of risdiplam treatment in younger children with SMA using the CEDAS. Gorni K, Baranello G, Conway E, Li Y, Kalache N.
118. Drug repurposing of olodaterol and forskolin for congenital myasthenic syndromes. Choueiri C, Ho K, Spendiff S, Meyer S, Grey M, Lochmüller H.
119. AAV-mediated gene editing strategies to treat Collagen VI-related Myopathies. Lin KH, Resner E, Maier M, Wagers A.
120. FREEDOM-DM1: A Phase 1, placebo-controlled single ascending dose study to evaluate PGN-EDODM1 in people with myotonic dystrophy type 1 (DM1). Hamel J, Brisson J, Wheeler T, Lochmuller H, Sampson J, Goyal NA, Phan H, Johnson N, Statland J, Lilleker J, Turner C, Pfeffer G, Shoskes J, Garg B, Song G, Lonkar P, Babcock S, Batra S, Yu S, Fraser P, Streck P, Larkindale J, McArthur J.  
121. Characterization of FDA-approved drugs that target the RNA-binding protein Staufen1 and mitigate the atrophy of skeletal muscle fibers following denervation. Charron M, Patel D, Almasi S, Chen H, Ravel-Chapuis A, Jasmin B.
122. The involvement of PARylation in muscle satellite cell fate and regeneration in a mouse model of muscular dystrophy. Della Giustina A, Saraci K, Menzies KJ.
123. Parylation peril: how poly-ADP-ribosylation affects satellite cell fate and regeneration. Saraci K, Green A, Niyonambaza S, Giustina AD, Menzies KJ.
124. High-Throughput 3-D Myobundle Platform Combining Cyclic Stretch and Electrical Pacing for Eccentric-Contraction Drug Screening in Duchenne Muscular Dystrophy. Ouellet J, Green A, Menzies K.
125. Multi-Omics Analysis Reveals PARylation as a Regulator of Muscle Health in Models of Obesity and Cancer-Cachexia. Nik-Akhtar A, Green A, Pandey D, Saraci K, Ouellet J, Cariaco Y, Bainbridge S, Menzies K.
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130. ATAD2: A Novel Regulator of Muscle Cell Proliferation, Differentiation, and Autophagy. Sedraoui S, Moamer A, Chaffer TJ, Mayaki D, Leduc-Gaudet J-P, Sandri M, Gouspillou G, Hussain S.
131. Single-AAV-mediated adenine base editing efficiently restores dystrophin expression and muscle function in mdx 4cv mice. Lam A, Lin K-H, Maier M, van Ooijen S, Ellis R, Messemer K, Wagers AJ. 
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133. Single AAV vector base editing rescues a nonsense variant in a humanized mouse model of Duchenne muscular dystrophy. Marks RM, Fatehi S, Khazeeva E, Bell E, Truong HA, Chi L, Yan B, Huynh E, Kozman N, Rok MJ, Delgado-Olguín P, Cohn RD, Ivakine EI.
134. Early dynamic functional neuromuscular reinnervation and maturation. Alvado S, Lafont A, Robitaille R.
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