Monday, Monday, September 12, 2022
OPENING-CEREMONY 5:30 PM > 6:00 PM Opening ceremony Apollon Auditorium
  • AFM-Téléthon President : L. Laurence TIENNOT-HERMENT (Evry, France)
  • Myology Presidents : E. Elizabeth MCNALLY (Chicago, USA), S. Shahragim TAJBAKHSH (Paris, France)
OPENING-LECTURE 6:00 PM > 6:45 PM Opening Lecture Apollon Auditorium
6:00 PM OL Story(ies) of astronomy on the French Riviera > E. Eric LAGADEC (Nice, France)
MEET-AND-GREET 7:00 PM > 9:00 PM Meet and greet time at the conference center Rhodes Room
Tuesday, Tuesday, September 13, 2022
13-AM-Plenary 8:30 AM > 10:00 AM Development, regeneration & ageing (part 1) Apollon Auditorium
  • Moderator : G. Gillian BUTLER-BROWNE (Paris, France)
8:30 AM S2L1 Molecular and functional diversity among satellite cell populations provides insights into disease > S. Shahragim TAJBAKHSH (Paris, France) 9:00 AM S2L2 Notch signaling: an emerging therapeutic target for muscle wasting diseases > Y. Yusuke ONO (Kumamoto, Japan) 9:30 AM S2L3 Myofiber syncytium - many identities in one cell > M. Minchul KIM (Illkirch-Graffenstaden, France) 9:45 AM S2L4 Dissecting the regulatory program controlling trapezius muscle development at the head trunk interface > C. Camille DUMAS (Marseille, France)
13-AM-Coffee 10:00 AM > 10:30 AM Coffee break and visit of the exhibit hall and poster room Rhodes Room
13-AM-Parallel-2 10:30 AM > 12:00 PM Development, regeneration & ageing (part 2) Apollon Auditorium
  • Moderator : F. Fabrice CHRETIEN (Paris, France)
10:30 AM S4L1 Engineering human stem cells for advanced neuromuscular disease and therapy modelling > F. Francesco Saverio TEDESCO (London, UK) 11:00 AM S4L2 LSD1-mediated demethylation of beta-catenin regulates muscle stem cell self-renewal potential > D. Delia CICCIARELLO (Lyon, France) 11:15 AM S4L3 Defective dystrophic thymus determines degenerative changes in skeletal muscle > Y. Yvan TORRENTE (Milano, Italy) 11:30 AM S4L4 USP18 a novel regulator of muscle cell differentiation and maturation, potentially regulating regeneration in dermatomyositis > V. Vered RAZ (Leiden, The Netherlands) 11:45 AM S4L5 Satellite Cell Dynamics in Growth and Regeneration of Skeletal Muscle in Normal and Dystrophic Mice > T. Terence PARTRIDGE (London, UK)
13-AM-Parallel-1 10:30 AM > 12:00 PM Lessons from the pandemic Athena Auditorium
  • Moderator : G. Guilhem SOLE (Bordeaux, France)
10:30 AM S3L1 COVID-19 and Myositis-a relevant problem ? > W. Werner STENZEL (Berlin, Germany) 11:00 AM S3L2 Telemedicine to overcome barriers in NMD- myths and realities > G. Gabriele SICILIANO (Pisa, Italy) 11:30 AM S3L3 Evaluation of anti-SARs-CoV-2 vaccination efficacy in patients with severe neuromuscular diseases > A. Alexia DAMOUR (Bordeaux, France) 11:45 AM S3L4 Safety of COVID-19 vaccination in a large French cohort of neuromuscular patients: data from the VACNEMUS study > M. Marlène BARNAY (Bordeaux, France)
13-AM-SYMPO 12:00 PM > 1:00 PM Industry symposium - Argenx: The gMG treatment individualization evolution: Implications for your patients Hermès Auditorium
12:00 PM SYMP1L1 Welcome and introduction > F. Francesco SACCÀ (Geneve) 12:10 PM SYMP1L2 Targeted treatments in gMG: Evolution of the treatment landscape and novel therapeutic targets > S. Suraj MULEY (Geneve, Switzerland) 12:25 PM SYMP1L3 Adapting the strategy: Working towards treatment individualization > F. Francesco SACCÀ (Geneve) 12:35 PM SYMP1L4 Panel discussion: Interactive case studies and practical implications for your patients 12:55 PM SYMP1L5 Q&A and close > F. Francesco SACCÀ (Geneve)
13-AM-LUNCH 12:00 PM > 2:00 PM Lunch break and visit of the exhibit hall and poster room
13-PM-YOUNG 2:00 PM > 3:00 PM Young Investigators Symposium Apollon Auditorium
  • Moderators : P. Pascale BOMONT (Lyon, France), T. Thierry TOURSEL (Evry, France)
2:00 PM S5L1 Delivery and expression of large dystrophins using adeno-associated viral vector and protein trans-splicing mediated by split inteins > H. Hichem TASFAOUT (Seattle, USA) 2:15 PM S5L2 Novel AAV capsid variant for muscle-directed gene therapy > J. Juliette LEMOINE (Evry, France) 2:30 PM S5L3 Inactivating the lipid kinase activity of PI3K-C2β is sufficient to rescue X-linked myotubular myopathy in mice > M. Marie GORET (Illkirch, France) 2:45 PM S5L4 PARP3 promotes myogenic differentiation and skeletal muscle function in cooperation with the histone methyltransferase EZH2. > Z. Zuleyha YILDIRIM (Illkirch, France)
13-PM-PLENARY 3:00 PM > 4:00 PM Advances and challenges in gene therapies (part 1) Apollon Auditorium
  • Moderator : S. Serge BRAUN (Evry, France)
3:00 PM S6L1 Challenges of AAV Gene Therapy Development for Duchenne Muscular Dystrophy > F. Francesco MUNTONI (London, UK) 3:20 PM S6L2 The management of safety of gene therapy of neuromuscular diseases, an emblematic example > S. Serge BRAUN (Evry, France) 3:40 PM S6L3 The liver as a model tissue to develop innovative solutions to the existing limitations of AAV gene therapy > G. Giuseppe RONZITTI (Evry, France)
13-PM-Coffee 4:00 PM > 4:30 PM Coffee break and visit of the exhibit hall and poster room Rhodes Room
13-PM-Parallel-1 4:30 PM > 6:00 PM Motor neuron diseases Apollon Auditorium
  • Moderator : C. Claude DESNUELLE (Nice, France)
4:30 PM S7L1 The role of DNA epigenetics in modulating Spinal Muscular Atrophy > P. Piera SMERIGLIO (Paris, France) 5:00 PM S7L2 Combination of BIO101 with antisense oligonucleotide therapy demonstrates synergistic beneficial effects in severe SMA-like mice > C. Cynthia BEZIER (Paris, France) 5:20 PM S7L3 Spinal cord MRI for early detection of presymptomatic pathology in C9orf72-mutation carriers: a longitudinal neuroimaging study. > G. Giorgia QUERIN (Paris, France) 5:40 PM S7L4 Extracellular Vesicle Sphingomyelins as Diagnostic Biomarkers in Motor Neurone Diseases > S. Stephanie DUGUEZ (Londonderry, UK)
13-PM-Parallel-2 4:30 PM > 6:00 PM Development, regeneration & ageing (part 3) Athena Auditorium
  • Moderators : E. Eric GILSON (Nice, France), S. Shahragim TAJBAKHSH (Paris, France)
4:30 PM S8L1 Dissecting stem cell regulation in skeletal muscle regeneration and aging through single - cell transcriptomics > B. Benjamin COSGROVE (Ithaca, USA) 5:00 PM S8L2 Preliminary results of a multiparametric quantitative NMR ageing study at rest and during exercise in the lower leg in healthy subjects between 20 and 65 years of age. > A. Alfredo LOPEZ KOLKOVSKY (Paris, France) 5:15 PM S8L3 The histone variant H2A.Z prevents accumulation of DNA damage and acts as a gatekeeper for skeletal muscle aging > L. Laurent SCHAEFFER (Lyon, France) 5:30 PM S8L4 The spectrum of IL-6 actions in healthy and bad aging and in muscle diseases > A. Antonio MUSARÒ (Roma, Italy) 5:45 PM S8L5 Therapeutic approach based on GDF5 to counteract age-related muscle wasting > M. Massiré TRAORE (Paris, France)
Wednesday, Wednesday, September 14, 2022
14-AM-PLENARY 8:30 AM > 10:00 AM Cardiomyology Apollon Auditorium
  • Moderators : H. Helge AMTHOR (Montingy-le-Bretonneux), K. Karim WAHBI (Paris, France)
8:30 AM S9L1 A history of cardiomyology > D. Denis DUBOC (Paris, France) 9:00 AM S9L2 Establishing new models for dystrophic cardiomyopathy > E. Elizabeth MCNALLY (Chicago, USA) 9:30 AM S9L3 EV for cardiac repair: rationale and translational roadblocks > P. Philippe MENASCHÉ (Paris, France)
14-AM-Coffee 10:00 AM > 10:30 AM Coffee break and visit of the exhibit hall and poster room Rhodes Room
14-AM-Parallel-1 10:30 AM > 12:00 PM Myotonic syndromes Apollon Auditorium
  • Moderator : B. Bertrand FONTAINE (Paris, France)
10:30 AM S10L1 Decoy gene therapy for Myotonic Dystrophy > D. Denis FURLING (Paris, France) 11:00 AM S10L2 Clinical Trial readiness in DM1 and DM2 > F. Federica MONTAGNESE (Munich, Germany) 11:30 AM S10L4 Mutation-driven personalized therapy in non-dystrophic myotonia > J-F. Jean-François DESAPHY (Bari, Italy)
14-AM-Parallel-2 10:30 AM > 12:00 PM Muscle fibrosis Athena Auditorium
  • Moderator : B. Bruno PEAULT (Los Angeles, USA)
10:30 AM S11L1 Metabolic reprogramming of skeletal muscle by resident macrophages points to CSF1R inhibitors as muscular dystrophy therapeutics > F. Farshad BABAEIJANDAGHI (Vancouver) 11:30 AM S11L2 Adipogenic properties of Fibro-Adipogenic Precursor cells in normal, regenerating and DMD mouse skeletal muscle > G. Georgiana PANCI (Lyon, France) 11:45 AM S11L5 Muscle fibrosis: a vicious circle between human fibroadipogenic progenitors and muscle fibers > M. Mona BENSALAH (Paris, France)
14-AM-SYMPO 12:00 PM > 1:00 PM Industry Symposium - Roche: Heading to the future of clinical research in neuromuscular disorders Hermès Auditorium
12:00 PM SYMP2L1 Welcome and introductions > F. Francesco MUNTONI (London, UK) 12:05 PM SYMP2L2 Seeking a better understanding of emerging therapies in neuromuscular disorders > F. Francesco MUNTONI (London, UK) 12:15 PM SYMP2L3 Innovative outcome measures: effective and personalised assessments in neuromuscular disorders > C. Charlotte LILIEN (Oxford, UK) 12:30 PM SYMP2L4 Innovative biomarkers: evaluating neuromuscular fatigue > G. Guillaume MILLET (Saint Etienne, France) 12:45 PM SYMP2L5 Panel discussion and Q&A
14-AM-LUNCH 1:00 PM > 2:30 PM POSTER LUNCH BREAK organized by AFM-Telethon
14-PM-PLENARY 2:30 PM > 4:00 PM Advances and challenges in gene therapies (part 2) Apollon Auditorium
  • Moderator : O. Odile BOESPFLUG-TANGUY (Paris, France)
2:30 PM S12L1 X-linked myotubular myopathy and liver dysfunction > A. Ana BUJ-BELLO (Evry, France) 3:00 PM S12L2 Modelling and editing Duchenne Muscular Dystrophy > W-H. Wolfram-Hubertus ZIMMERMANN (Goettingen, Germany) 3:30 PM S12L3 Application of CRISPR/Cas9 strategy for gene therapy of Myotonic Dystrophy type 1 > G. Germana FALCONE (Monterotondo, Italy)
14-PM-Coffee 4:00 PM > 4:30 PM Coffee break and visit of the exhibit hall and poster room Rhodes Room
14-PM-Parallel-1 4:30 PM > 6:00 PM Neuromuscular junction Apollon Auditorium
  • Moderator : S. Shahram ATTARIAN (Marseille, France)
4:30 PM S13L1 New treatments for autoimmune myasthenia gravis > E. Emilien DELMONT (Marseille, France) 5:00 PM S13L2 Use of human pluripotent stem cells for Neuromuscular Disorders > C. Cecile MARTINAT (Evry, France) 5:30 PM S13L3 Targeting the neuromuscular junction (NMJ) to treat congenital myasthenic syndromes and inherited motor neuropathies with NMJ dysfunction > S. Sally SPENDIFF (Ottawa, Canada)
14-PM-Parallel-2 4:30 PM > 6:00 PM FSHD Athena Auditorium
4:30 PM S14L1 Facioscapulohumeral muscular dystrophy: clinical, genetic and nuclear heterogeneity from a single locus > S. Silvere VAN DER MAAREL (Leiden, The Netherlands) 5:00 PM S14L2 Inflammatory responses in FSHD > S. Sabrina SACCONI (Nice, France) 5:30 PM S14L3 Induced pluripotent stem cells for modeling neuromuscular disorders: the example of FacioScapuloHumeral Dystrophy > F. Frederique MAGDINIER (Marseille, France)
14-PM-ND 8:00 PM > 11:30 PM Networking dinner (not included in registration fees-separate registration required)
Thursday, Thursday, September 15, 2022
15-AM-PLENARY 8:30 AM > 10:00 AM New Concepts Apollon Auditorium
  • Moderator : G. Giorgio TASCA (Roma, Italy)
8:30 AM S15L1 Oculopharyngodistal myopathy > I. Ichizo NISHINO (Tokyo, Japan) 9:00 AM S15L2 Latest advances in CMT: Disease mechanisms and novel therapy concepts in demyelinating neuropathy > M. Michael SEREDA (Göttingen, Germany) 9:30 AM S15L3 The node of Ranvier: a vulnerable site for autoimmunity > L. Luis QUEROL (BARCELONA)
15-AM-Parallel-1 10:30 AM > 12:00 PM New Tools Apollon Auditorium
  • Moderator : K. Ketan PATEL (Reading)
10:30 AM S16L1 3D printed, anisotropic, and porous dense collagen hydrogels to mimic skeletal muscle extracellular matrix > M. Marie CAMMAN (Paris, France) 11:00 AM S16L2 The generation of a new preclinical rat model for DMD leads to the identification of the TSHR pathway as a regulator of senescence in muscle stem cells. > F. Frederic RELAIX (Créteil, France) 11:30 AM S16L3 A novel extrusion 3D bio-printing system for skeletal muscle tissue engineering. > S. Stefano TESTA (Marseille, France)
15-AM-Parallel-2 10:30 AM > 12:00 PM LGMD Athena Auditorium
  • Moderator : E. Elizabeth MCNALLY (Chicago, USA)
10:30 AM S17L1 The first standards of c are guidelines for a limb girdle muscular dystrophy > V. Volker STRAUB (Newcastle, UK) 11:00 AM S17L2 Genetic Profile of Patients with Limb-Girdle Muscle Weakness in the Chilean Population > J. Jorge BEVILACQUA (Santiago, Chili) 11:30 AM S17L3 Preclinical development of a gene therapy for three prevalent forms of LGMD > I. Isabelle RICHARD (Evry, France)
15-AM-SYMPO 12:00 PM > 1:00 PM Industry Symposium - UCB - Auto-immune myasthenia: epidemiological description of the French myasthenic population and its therapeutic management; patients' expectations concerning new therapies in MG Hermès Auditorium
  • Moderator : S. Shahram ATTARIAN (Marseille, France)
SYMPL1 Myasthenia gravis epidemiologic data first analysis of French Insurance health data base SNDS > E. Emmanuelle SALORT-CAMPANA (Marseille, France) SYMPL2 Patient’s voices: Which patient expectation for the new therapeutic option on going in myasthenia gravis  ?
15-AM-Lunch 1:00 PM > 2:00 PM Lunch break and visit of the exhibit hall and poster room
15-PM-PLENARY-1 2:00 PM > 3:00 PM Late Breaking News Apollon Auditorium
2:00 PM S18L1 Update on the development of Givinostat in DMD > P. Paolo Umberto BETTICA (Cinisello Balsamo, Italy) 2:15 PM S18L2 An AAV-ShRNA DUX4-based therapy to treat FacioScapuloHumeral muscular Dystrophy (FSHD) > J. Julie DUMONCEAUX (London, UK) 2:30 PM S18L3 CD38-NADase is a major contributor to DMD phenotype > S. Sabine DE LA PORTE (Montigny-le-Bretonneux, France) 2:45 PM S18L4 Severe mitochondrial dysfunctions caused by a heterozygous DES mutation in iPSC-derived cardiomyocytes > Y. Yeranuhi HOVHANNISYAN (Angers, France)
15-PM-PLENARY-2 3:00 PM > 5:00 PM Mitochondria and muscle Apollon Auditorium
  • Moderators : O. Olivier BARIS (Angers, France), H. Holger PROKISCH (Munich, Germany), R. Rita HORVATH (Cambridge, UK)
3:00 PM S19L1 Mechanisms of mitochondrial myopathy: mosaic stress responses, metabolic remodeling and curable NAD-deficiency > A. Anu SUOMALAINEN (Helsinki, Finland) 3:45 PM S19L2 Regulation of skeletal muscle bioenergetics through mitochondrial calcium during exercise and aging > J. Jerome FEIGE (Lausanne, Switzerland) 4:15 PM S19L3 Structural heterogeneity of the human respiratory chain > C. Cristina UGALDE (Madrid, Spain) 4:30 PM S19L4 OMA1 mediates local and global stress responses against protein misfolding in CHCHD10 mitochondrial myopathy > D. Derek NARENDRA (Bethesda, USA) 4:45 PM S19L5 Efficacy and genetic safety of deoxyribonucleosides as a therapy for mitochondrial DNA replication defects caused by mutations in genes involved in mtDNA maintenance > J. Javier RAMÓN (Barcelona, Spain)
15-PM-Coffee 5:00 PM > 5:30 PM Coffee break in Agora 2
15-PM-SYMPO 6:00 PM > 7:00 PM Symposium: Development of Deoxynucleoside Therapy for Thymidine Kinase 2 Deficiency (TK2d)  Apollon Auditorium
  • Moderator : M. Michio HIRANO (New York, USA)
SYMPL1 Clinical overview and Natural History Spectrum of TK2d > C. Caterina GARONE (Bologna, Italy) SYMPL2 Pharmacological and Gene Therapy in TK2 mutant mice  > C. Carlos LOPEZ-GOMEZ (Malaga, Spain) SYMPL3 Contributions of a Spanish Network to TK2d diagnosis and therapy > C. Cristina DOMÍNGUEZ-GONZÁLEZ (Madrid, Spain) SYMPL4 Expanded Access Deoxynucleos(t)ide TK2d Therapy at a US Site > M. Michio HIRANO (New York, USA)
PS1 7:00 PM > 8:00 PM Poster Session 1 Rhodes Room
Friday, Friday, September 16, 2022
16-AM-Plenary 8:30 AM > 10:00 AM Innate Immunity and Mitochondria Athena Auditorium
  • Moderators : V. Vincent PROCACCIO (Angers, France), A. Agnes ROTIG (Paris)
8:30 AM MITOS1L1 The integrated stress response in mitochondrial cardiomyopathy > T. Thomas LANGER (Cologne, Germany) 9:00 AM MITOS1L2 Immune mediated disease pathogenesis in Leigh syndrome > S. Simon JOHNSON (Seattle, USA) 9:30 AM MITOS1L3 Nuclear Sensing of Breaks in Mitochondrial DNA Enhances Immune Surveillance > M. Marco TIGANO (Philadelphia, USA) 9:45 AM MITOS1L4 Cell lineage-specific distribution of pathogenic mtDNA mutations in the immune system and purify selection during the adaptive immune response > J. Jingdian ZHANG (Stockholm, Sweden)
16-AM-Coffee 10:00 AM > 10:30 AM Coffee break and visit of the exhibit hall and poster room Rhodes Room
16-AM-Plenary-2 10:30 AM > 12:00 PM IPS cells and Organoids as alternative to mouse models ? Athena Auditorium
  • Moderators : M. Maria FALKENBERG (Mölndal, Sweden), V. Valeria TIRANTI (Milan, Italy)
10:30 AM MITOS2L1 Modelling OPA1 Dysfunction in iPSC-derived Retinal Ganglion Cells > J. Joshua HARVEY (Camberwell, UK) 11:00 AM MITOS2L2 Drug discovery of mitochondrial diseases using patient-specific brain organoids > A. Alessandro PRIGIONE (Düsseldorf, Germany) 11:30 AM MITOS2L3 PKAN stem cell derived neurons and astrocytes show massive iron accumulation mimicking the human phenotype. > S. Sonia LEVI (Roma, Italy) 11:45 AM MITOS2L4 Mitochondrial Regulation of Neural Stem Cell Ageing in Progressive Multiple Sclerosis > R-B. Rosana-Bristena IONESCU (Cambridge, UK)
16-PM-SYMPO 12:00 PM > 1:00 PM Optic neuropathies : From LHON to DOA Athena Auditorium
  • Moderator : R. Robert PITCEATHLY (London, UK)
12:00 PM MITOS3L1 Autosomal dominant optic atrophy > G. Guy LENAERS (Angers, France) 12:25 PM MITOS3L2 Leber hereditary optic neuropathy > P. Patrick YU-WAI-MAN (Cambridge, UK)
16-AM-Lunch 1:00 PM > 2:00 PM Lunch break and visit of the exhibit hall and poster room
16-PM-Plenary 2:00 PM > 3:30 PM Neurodegeneration and Mitochondria Athena Auditorium
  • Moderators : T. Timothy WAI (Paris, France), A. Antonio ZORZANO (Barcelona, Spain)
2:00 PM MITOS4L1 CHCHD10-related neurodegeneration: from gene to therapeutic approaches > V. Veronique PAQUIS-FLUCKLINGER (Nice, France) 2:30 PM MITOS4L2 Metabolic rewiring precedes OXPHOS dysfunction in a mutant CHCHD10 mouse model > G. Giovanni MANFREDI (New York, USA) 3:00 PM MITOS3L3 Mitochondrial dysfunction and Ca2+ dysregulation in COQ8A-Ataxia Purkinje neurons that can be rescued by CoQ10 treatment > H. Helene PUCCIO (Lyon, France) 3:15 PM MITOS4L4 Bi-allelic variants in TAMM41 are associated with low muscle cardiolipin levels leading to neonatal mitochondrial disease > R. Rob TAYLOR (Newcastle, UK)
16-PM-Coffee 3:30 PM > 4:00 PM Coffee break and visit of the exhibit hall and poster room Rhodes Room
16-PM-Plenary-2 4:00 PM > 5:30 PM New models for genotype/phenotype correlation Athena Auditorium
  • Moderators : R. Robert MCFARLAND (Newcastle, UK), S. Shamima RAHMAN (London, UK)
4:00 PM MITOS5L1 Mechanisms and logic of mitochondrial metabolite signaling > E. Edward CHOUCHANI (Boston, USA) 4:30 PM MITOS5L2 Modelling phenotypes of mitochondrial translation defects in vitro and in vivo > R. Rita HORVATH (Cambridge, UK) 5:00 PM MITOS5L3 Mitochondrial genome engineering in vivo > M. Michal MINCZUK (Cambridge, UK) 5:15 PM MITOS5L4 The first in human clinical application of autologous mesoangioblasts as cell therapy medical product in m.3243A>G mutation carriers > F. Florence VAN TIENEN (Maastricht, The Netherlands)
16-PM-Coffee 5:00 PM > 5:30 PM Coffee break and Visit of the exhibit hall and poster room Rhodes Room
16-PM-Plenary-3 5:30 PM > 7:00 PM E-MIT Meeting : European Mitochondrial Society Athena Auditorium
  • Moderators : V. Valerio CARELLI (Bologna, Italy), V. Valeria TIRANTI (Milan, Italy)
5:30 PM MITOS6L1 Introductory talk > M. Massimo ZEVIANI (Padova, Italy) 6:30 PM MITOS6L3 General discussion
PS2 7:00 PM > 8:00 PM Poster Session 2 Rhodes Room
16-PM-NE 8:00 PM > 10:30 PM Networking Event Rhodes Room
Saturday, Saturday, September 17, 2022
17-AM-Plenary 8:30 AM > 10:00 AM New treatments for mitochondrial diseases: update on recent and current clinical trials Athena Auditorium
  • Moderators : P. Patrick CHINNERY (Cambridge, UK), T. Thomas KLOPSTOCK (München, Germany)
8:30 AM MITOS7L1 Clinical trials landscape in primary mitochondrial diseases in the USA: looking into the future > A. Amel KARAA (Boston, USA) 8:45 AM MITOS7L2 Sonlicromanol in primary mitochondrial disease MELAS spectrum disorders > J. Jan SMEITINK (Beuningen, The Netherlands) 9:00 AM MITOS7L3 Mitochondrial disease clinical trials: experience gained at the London highly specialised service for rare mitochondrial disorders > R. Robert PITCEATHLY (London, UK) 9:15 AM MITOS7L4 Elamipretide treatment and Barth syndrome: combined data from a phase 2/3 clinical trial and a natural history comparison study > H. Hilary VERNON (Baltimore, USA) 9:30 AM MITOS7L5 General discussion between the panel and the audience
17-AM-Coffee 10:00 AM > 10:30 AM Coffee break and visit of the exhibit hall and poster room Rhodes Room
17-AM-Plenary-2 10:30 AM > 12:15 PM Flash session with hot topics Athena Auditorium
  • Moderators : G. Giovanni MANFREDI (New York, USA), A. Anu SUOMALAINEN (Helsinki, Finland)
10:30 AM MITOS8L1 Adapting DNA editing enzymes to treat mitochondrial disease > C. Carlos MORAES (Miami, USA) 10:45 AM MITOS8L2 Inhibition of mtDNA transcription has beneficial effects on metabolism > N-G. Nils-Göran LARSSON (Stockholm, Sweden) 11:00 AM MITOS8L3 About Neural stem cell trafficking and secretion of functional mitochondria via extracellular vesicles > S. Stefano PLUCHINO (Cambridge, UK) 11:15 AM MITOS8L4 Targeting metabolism to purge mutant mitochondrial DNAs > A. Antonella SPINAZZOLA (London) 11:30 AM MITOS8L5 Genetic variants affecting NQO1 protein levels impact on efficacy of idebenone treatment in Leber’s hereditary optic neuropathy > V. Valerio CARELLI (Bologna, Italy) 11:45 AM MITOS8L6 Neuroglobin overexpression in cerebellar neurons of Harlequin mice improves mitochondrial robustness and reduces ataxic behavior > M. Marisol CORRAL-DEBRINSKI (Paris, France) 12:00 PM MITOS8L7 Double administration of self-complementary AAV9NDUFS4 prevents Leigh disease in Ndufs4−/− mice > C. Carlo VISCOMI (Padova)
17-PM-CLOSING 12:15 PM > 1:00 PM Closing Ceremony Athena Auditorium
  • Chairman : V. Veronique PAQUIS-FLUCKLINGER (Nice, France)
  • Speakers : AFM REPRESENTATIVE, V. Valerio CARELLI (Bologna, Italy), V. Vincent PROCACCIO (Angers, France), V. Valeria TIRANTI (Milan, Italy)
12:15 PM MITOS9L1 Best MitoNice poster Award 12:30 PM MITOS9L2 Closing remarks

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