Monday, 22 April 2024
S1 17:00 > 19:00 OPENING CEREMONY AMPHITHEATRE BORDEAUX
17:00 S1L1 Welcome ceremony > AFM-Téléthon President> Myology Presidents > L. Laurence TIENNOT-HERMENT (Paris, France), J. John VISSING (Copenhaghen, Denmark), A. Annemieke AARTSMA-RUS (Leiden, The Netherlands) 17:30 S1L2 Opening Lecture 1 : Nucleoside-modified mRNA-LNP therapeutics > D. Drew WEISSMAN (Philadephia, USA) 18:15 S1L3 Opening Lecture 2 : The road to gene therapy for DMD > J. Jeffrey CHAMBERLAIN (Seattle, USA)
B1 19:00 > 21:00 MEET AND GREAT TIME Hall Havane AB
Tuesday, 23 April 2024
S2 08:30 > 10:00 PLENARY SESSION // MYOGENESIS AMPHITHEATRE BORDEAUX
  • Chairmen : B. Bénédicte CHAZAUD (Lyon, France), F. Fabrice CHRÉTIEN (Paris, France)
08:30 S2L1 Muscle stem and niche cell dynamics in pathology > S. Shahragim TAJBAKHSH (Paris, France) 09:00 S2L2 > J. Jérôme FEIGE (Lausanne, Switzerland) 09:30 S2L3 The histone variant H2A.Z is required for DNA repair in muscle fibers and prevents premature aging > E. Edwige BELOTTI (Lyon, France) 09:45 S2L4 Contribution of Insulin-Like Growth Factor-I secreted from Fibro-Adipogenic Progenitors to Skeletal Muscle Regeneration > Y. Yangyi LUO (Gainesville, USA)
B2 10:00 > 10:30 COFFEE BREAK AND VISIT OF THE EXHIBIT HALL AND POSTER ROOM Hall Havane AB
S3 10:30 > 12:00 PARALLEL SESSION 1 // MUSCLE FIBROSIS / AGEING AMPHITHEATRE BORDEAUX
  • Chairman : G. Gillian BUTLER-BROWNE (Paris, France)
10:30 S3L1 > R. Rachelle CROSBIE (Los Angeles, USA) 11:00 S3L2 > M. Melissa SPENCER (Los Angeles, USA) 11:30 S3L3 Targeting endoplasmic reticulum and mitochondria to resolve protein amyloid deposits in oculopharyngeal muscular dystrophy > C. Capucine TROLLET (Paris, France) 11:45 S3L4 Targeting FAP to reduce fibrosis in DMD: a rationale for a preclinical therapeutic trial in the GRMD dog model. > I. Inès BARTHÉLÉMY (Paris, France)
S4 10:30 > 12:00 PARALLEL SESSION 2 // CONGENITAL MYOPATHIES AMPHITHEATRE HAVANE
  • Chairman : I. Isabelle MARTY (Grenoble, France)
10:30 S4L1 Congenital myopathies > J. James DOWLING (Toronto, Canada) 11:00 S4L2 Congenital myopathies in adulthood > N. Nicol VOERMANS (Nijmegen, Netherlands) 11:30 S4L3 ASC-1 related myopathy reveals transcriptional co-activation as a new regulator of myogenesis: expanding the molecular spectrum and identifying the mechanisms of an emerging congenital myopathy. > A. Ana FERREIRO (Paris, France) 11:45 S4L4 Ultrarare CACNA1S congenital-myopathies pharmacological treatment through HiPSC-based Drug repurposing > R. Reem BOU AKAR (Créteil, France)
SYMPO1 12:00 > 13:00 INDUSTRY SYMPOSIUM // SAREPTA
SYMPO2 13:00 > 14:00 INDUSTRY SYMPOSIUM // LUPIN
B3 13:00 > 14:00 LIGHT LUNCH BUFFET (OFFERED) AND VISIT OF THE EXHIBIT HALL AND POSTER ROOM Hall Havane AB
S5 14:00 > 15:00 YOUNG INVESTIGATORS SYMPOSIUM AMPHITHEATRE BORDEAUX
  • Chairmen : S. Serge BRAUN (Evry, France), T. Thierry TOURSEL (Evry, France)
14:00 S5L1 Muscle targeted gene therapy to cure BIN1 centronuclear myopathy > J. Jacqueline JI (Illkirch-Graffenstaden, France) 14:15 S5L2 RNA-sequencing of Type II SMA paravertebral muscle after treatment reveals distinct molecular subtypes. > F. Fiorella GRANDI (Paris, France) 14:30 S5L3 The damaged lysosome is a therapeutic target for combined therapy in Duchenne muscular dystrophy > A. Abbass JABER (Evry, France) 14:45 S5L4 Deciphering the consequences of SORBS1 mis-splicing in myotonic dystrophy type 1 > M. Morgan GAZZOLA (Corbeil-Essonnes, France)
S6 15:00 > 15:20 LATE BREAKING NEWS AMPHITHEATRE BORDEAUX
  • Chairman : S. Serge BRAUN (Evry, France)
15:00 S6L1
RT 15:20 > 16:00 Duchenne gene therapy: lessons learned and future directions - Round table discussion AMPHITHEATRE BORDEAUX
  • Chairman : A. Annemieke AARTSMA-RUS (Leiden, The Netherlands)
  • Speakers : J. Jeff CHAMBERLAIN (Seattle, USA), E. Eugenio MERCURI (Rome, Italy), F. Francesco MUNTONI (London, United Kingdom)
B4 16:00 > 16:30 COFFEE BREAK AND VISIT OF THE EXHIBIT HALL AND POSTER ROOM Hall Havane AB
S7 16:30 > 18:00 PARALLEL SESSION 1 // SMA / NOVEL THERAPIES AMPHITHEATRE BORDEAUX
  • Chairman : J. Judith MELKI (Le Kremlin-Bicetre, France)
16:30 S7L1 New treatment in Spinal Muscular Atrophy > L. Laurent SERVAIS (Oxford, UK) 17:00 S7L2 Improved understanding of SMA and development of further treatment strategies > A. Arthur BURGHES (Columbus OH, United States) 17:30 S7L3 Bi-regional newborn screening program for spinal muscular atrophy in France linked to a nationwide strategy for standardized treatment and follow-up > V. Vincent LAUGEL (Strasbourg, France) 17:45 S7L4 LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates SBMA phenotypes in flies and mice. > R. Roberta ANDREOTTI (Padova, Italy)
S8 16:30 > 18:00 PARALLEL SESSION 2 // FSHD AMPHITHEATRE HAVANE
  • Chairmen : S. Sabrina SACCONI (Nice, France), S. Silvere VAN DER MAAREL (Leiden, The Netherlands)
16:30 S8L1 FSHD: Clinical Manifestation and Therapeutic Prospects > R. Rabi TAWIL (Rochester, USA) 17:00 S8L2 Decoding DUX4: understanding a trendy landscape of FSHD pathogenesis > A. Alberto ROSA (Cordoba, Argentina) 17:30 S8L3 Exchange of subtelomeric regions between chromosomes 4q and 10q: a novel way to revert the FSHD genotype and phenotype > A. Anna SCHWAGER (Villejuif, France) 17:45 S8L4 Targeting muscle degeneration in FSHD: insights from patients’ muscle stem cell niche to unlock disease mechanisms > L. Lorena DI PIETRO (Rome, Italy)
SYMPO3 18:00 > 19:00 INDUSTRY SYMPOSIUM // EDGEWISE
Wednesday, 24 April 2024
S9 08:30 > 10:00 PLENARY SESSION // MUSCLE, SPORT, EXERCICE AMPHITHEATRE BORDEAUX
  • Chairmen : X. Xavier BIGARD (Ugine, France), N. Nicoline VOET (Wageningen, The Netherlands)
08:30 S16L1 > M. Michael KJAER (Denmark) 09:00 S16L2 > J. John VISSING (Copenhaghen, Denmark) 09:30 S16L3 Muscle-to-brain communication under voluntary and forced exercise training applied in the context of obesity in mice. > A. Antoine DELPIERRE (Mons, Belgium) 09:45 S16L4 Thyroid hormone regulates skeletal muscle physiology and affects physical exercise efficiency > M. Monica DENTICE (Naples, Italy)
B5 10:00 > 10:30 COFFEE BREAK AND VISIT OF THE EXHIBIT HALL AND POSTER ROOM Hall Havane AB
S10 10:30 > 12:00 PARALLEL SESSION 1 //CMT & NEUROPATHIES AMPHITHEATRE BORDEAUX
  • Chairman : S. Shahram ATTARIAN (Marseille, France)
10:30 S10L1 Current treatments and new perspectives in Charcot-Marie-Tooth neuropathies > D. Davide PAREYSON (Milano, Italy) 11:00 S10L2 Charcot-Marie-Tooth disease: an overview of genetic and phenotypic variability > T. Tanya STOJKOVIC (PARIS, France) 11:30 S10L3 SCREEN4PN: Efficient Evaluation Of Therapeutic Compounds For Charcot-Marie-Tooth Disease Using Patient-Derived Induced Motor Neurons and Neuromuscular Organoids > V. Vincent TIMMERMAN (Antwerpen, Belgium) 11:45 S10L4 Revisit Of Patients With Phenotypic Hereditary Polyneuropathy Without Genetic Confirmation > L. Louise Sloth KODAL (Copenhagen, Denmark)
S11 10:30 > 12:00 PARALLEL SESSION 2 // LGMD AMPHITHEATRE HAVANE
  • Chairman : J. Jorge A. BEVILACQUA (Santiago, Chili)
10:30 S11L1 > J. Jordi DIAZ-MANERA (Newcastle Upon Tyne, UK) 11:00 S11L2 Development of gene therapy for three prevalent forms of LGMD > I. Isabelle RICHARD (Evry-Courcouronnes, France) 11:30 S11L3 Biallelic variants in SNUPN cause a limb girdle muscular dystrophy with myofibrillar-like features > A. Alberto DAMBORENEA (Donostia-San Sebastián, Spain) 11:45 S11L4 Unraveling a Novel Form of Muscular Dystrophy Caused by Recessive SNUPN Mutations > M. Marwan NASHABAT (Istanbul, Turkey)
SYMPO4 12:00 > 13:00 INDUSTRY SYMPOSIUM // ITALFARMACO
B6 13:00 > 14:30 LIGHT LUNCH (OFFERED) AND VISIT OF THE EXHIBIT HALL Hall Havane AB
SYMPO5 13:30 > 14:30 INDUSTRY SYMPOSIUM // PFIZER
S12 14:30 > 16:00 PLENARY SESSION // POSTER FLASH SESSION AMPHITHEATRE BORDEAUX
  • Chairmen : E. Edoardo MALFATTI (Paris, France), F. France PIETRI-ROUXEL (Paris, France)
14:30 S12L1 15:00 S12L2 15:30 S12L3
S13 16:00 > 17:30 PARALLEL SESSION 1 // OUTCOME MEASURES / PHYSIOTHERAPIES AMPHITHEATRE BORDEAUX
  • Chairman : E. Erik NIKS (Leiden, The Netherlands)
16:00 S13L1 Natural history studies : challenges and limitations > J-Y. Jean-Yves HOGREL (Paris, France) 16:30 S13L2 Natural history studies and clinical trial readiness: successes and future directions > L. Lindsay ALFANO (Columbus, USA) 17:00 S13L3 The validity of cardiopulmonary exercise testing for assessing aerobic capacity in neuromuscular diseases > E. Eric VOORN (Amsterdam, Netherlands) 17:15 S13L4 Longitudinal quantitative MRI of forearm and arm muscles in dysferlinopathy > H. Harmen REYNGOUDT (Paris, France)
S14 16:00 > 17:30 PARALLEL SESSION 2 // TITINOPATHIES AMPHITHEATRE HAVANE
  • Chairman : B. Bjarne UDD (Helsinki, Finland)
16:00 S14L1 Clinical Interpretation of Titin Variants > M. Marco SAVARESE (Helsinki, Finland) 16:30 S14L2 Recessive titinopathy: recently emerged ‘titan’of the paediatric muscle disease world > E. Emily OATES (Sydney, Australia) 17:00 S14L3 Personalized genetic counselling in titinopathies: exon usage as the key to gain prognostic insights in the whole clinical spectrum > M. Maria Francesca DI FEO (Genova, Italy) 17:15 S14L4 Distal upper limb onset titinopathy evolves towards a hereditary myopathy with early respiratory failure (HMERF) phenotype. > M. Mario FUENTEALBA (Concepcion, Chile)
SYMPO6 17:30 > 18:30 INDUSTRY SYMPOSIUM // ALEXION
ND 20:00 > 20:00 NETWORKING DINNER / BATEAUX-MOUCHES PARIS
Thursday, 25 April 2024
S15 08:30 > 10:00 PLENARY SESSION // GENE EDITING AMPHITHEATRE BORDEAUX
  • Chairman : A. Annemieke AARTSMA-RUS (Leiden, The Netherlands)
08:30 S15L1 > C. Charles GERSBACH (Durham, USA) 09:00 S15L2 > R. Richard HORGAN (Woodbridge, CT, USA) 09:30 S15L3 Human iPS-based disease models and CRISPR-Cas technology for the development of genetic therapies for LMNA­-related skeletal muscle laminopathies > D. Daniel MOORE (London, UK) 09:45 S15L4 First correction by Prime editing of mutations in the RYR1 gene > J. Jacques P. TREMBLAY (Quebec, Canada)
B7 10:00 > 10:30 COFFEE BREAK AND VISIT OF THE EXHIBIT HALL AND POSTER ROOM Hall Havane AB
S16 10:30 > 12:00 PARALLEL SESSION 1 // METABOLIC DISORDERS AMPHITHEATRE BORDEAUX
  • Chairman : C. Claude DESNUELLE (Nice, France)
10:30 S16L1 > P. Pascal LAFORET (Paris, France) 11:00 S16L2 New facets of Pompe disease > B. Benedikt SCHOSER (Munich, Germany) 11:30 S16L3 Decoding the transcriptome of late-onset Pompe disease muscle at single nucleus resolution > A. Alexandra MONCEAU (Newcastle-upon-Tyne) 11:45 S16L4 Abnormal autophagy is a critical mechanism in TANGO2-related rhabdomyolysis > H. Hortense DE CALBIAC (Paris, France)
S17 10:30 > 12:00 PARALLEL SESSION 2 // INFLAMMATORY MYOPATHIES AMPHITHEATRE HAVANE
10:30 S17L1 > O. Olivier BENVENISTE (Muenchen, Germany) 11:00 S17L2 > I. Ichizo NISHINO (Kodaira, Japan) 11:30 S17L3 Absence of gene pathogenic variants and strong association with HLA-DRB1*11:01 in statin-naïve early-onset anti-HMG-CoA reductase necrotizing myopathy > L. Laura LLANSO CALDENTEY (Barcelona, Spain) 11:45 S17L4 Proteomic profiling to define IIM-associated marker proteins in cultured human myoblasts > A. Andreas ROOS (Dusseldorf, Germany)
SYMPO7 12:00 > 13:00 INDUSTRY SYMPOSIUM // UCB
B8 13:00 > 14:30 LIGHT LUNCH BUFFET (OFFERED) AND VISIT OF THE EXHIBIT HALL Hall Havane AB
S18 14:30 > 16:00 PARALLEL SESSION 1 // NEUROMUSCULAR JUNCTION AMPHITHEATRE BORDEAUX
  • Chairman : P. Pedro RODRIGUEZ (Dakar, Senegal)
14:30 S18L1 > J. Jan VERSCHUUREN (Amsterdam, The Netherlands) 15:00 S18L2 Moving towards personalized medicine in Myasthenia Gravis through biomarkers and the power of physical exercise > A. Anna PUNGA (Uppsala, Sweden) 15:30 S18L3 Origin of the thymic interferon type I signature in myasthenia Gravis: role of endogenous nucleic acids > R. Rozen LE PANSE (Paris, France) 15:45 S18L4 Concomitant Corticosteroid Use in Ravulizumab-Treated Adults With Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Final Results From the Phase 3 CHAMPION MG Open-label Extension > D. Djillali ANNANE (Paris, France)
S19 14:30 > 16:00 PARALLEL SESSION 2 // MUSCLE AND MITOCHONDRIA AMPHITHEATRE HAVANE
  • Chairman : V. Vincent PROCACCIO (Angers, France)
14:30 S19L1 Mitochondrial medicine: from mechanisms to treatments > R. Rita HORVATH (Cambridge, UK) 15:00 S19L2 > T. Thomas KLOPSTOCK (Muenchen, Germany) 15:30 S19L3 Mitochondrial Myopathy caused by TK2 deficiency: lessons from a large Brazilian cohort. > C. Christiane ARAUJO (Sao Paolo, Brazil) 15:45 S19L4 Dissection of mechanisms underlying the pathogenesis of Duchenne Muscular Dystrophy > E. Elena CANNONE (Brescia, Italy)
S20 16:00 > 16:15 YOUNG RESEARCHERS AWARDS CEREMONY AMPHITHEATRE BORDEAUX
  • Chairman : T. Thierry TOURSEL (Evry, France)
S21 16:15 > 17:15 LATE BREAKING NEWS AMPHITHEATRE BORDEAUX
16:15 S21L1 Robust preclinical data support development of DYNE-251 as a potential treatment for individuals with DMD mutations amenable to exon 51 skipping > E. Erwan DELAGE (Paris, France) 16:30 S21L2 Striking cardioprotective effects of AdipoRon in an aged mouse model of Duchenne Muscular Dystrophy > M. Michel ABOU-SAMRA (Brussels, Belgium) 16:45 S21L3 Optimising a multimodal pathway to discover genes involved in Satellite Cell-opathies: neuromuscular disorders caused by muscle stem cell dysfunction > P. Peter ZAMMIT (London, United Kingdom) 17:00 S21L4
S22 17:15 > 17:30 CLOSING CEREMONY AMPHITHEATRE BORDEAUX
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