2025
Aartsma-Rus, A., “From rare to nanorare: applying lessons learned from Duchenne RNA therapy to individualized treatments”, Heinrich Wieland Prize Award Symposium, 11 December 2025, Munich, Germany.
Aartsma-Rus, A., “N-of-1 therapy development for muscular dystrophies: yes or no?”, World Muscle Society Conference Debate, 7-11 October 2025, Vienna, Austria.
Aartsma-Rus, A., “Optimizing therapy development for Duchenne and other rare diseases”, Scilife conference, 1 October 2025, Stockholm, Sweden.
Aartsma-Rus, A., “Optimizing exon skipping for Duchenne muscular dystrophy and The N=1 Collaborative”, Symposium of the 10th anniversary of the NATJS, 2-3 July 2025, Kobe, Japan.
Aartsma-Rus, A., “Developing N=1 treatments for patients with unique variants.” German Society of Human Genetics meeting, 2-5 April 2025, Innsbruck, Austria.
Aartsma-Rus, A., “Developing individualized oligonucleotide treatments.” Cold Spring Harbor Laboratory meeting on nucleic acid therapeutics, 19-22 March 2025, Cold Spring Harbor, NY, USA.
Aartsma-Rus, A., “From Duchenne exon skipping to N=1 treatment: Applying lessons learned for (very) rare disease patients.” Swiss RNA Therapeutics Summit, 23-24 January 2025, Bern, Switzerland.
Ahsan, I., Synofzik, M., “Development and Efficacy Testing of exon-skipping Antisense Oligonucleotide (ASO): a systematic platform approach for Ataxia-Telangiectasia”, Dutch Antisense Therapeutics Symposium, 12-13 June 2025, Groningen (poster).
Andrés-Santamaría, R., “Comparative Safety Assessments of N-of-Few Antisense Oligonucleotides in Rats”, Annual Meeting of the OTS Oligonucleotide Therapeutics Society, 19-22 October 2025, Budapest, Hungary (poster).
Andrés-Santamaría, R., “Development of a safety pipeline for n-of-1 ASOs in rats”, 4th Dutch Antisense Therapeutics Symposium, 12-13 June 2025, University Medical Center Groningen, Groningen, the Netherlands (poster).
Andrés-Santamaría, R., “Development and Validation of a Safety Pipeline for N-of-1 ASOs in Rats”, Medical-Genetic Center South-West Netherlands (MGC) PhD Student Workshop, 2-5 June 2025, DJH Youth Hostel, Düsseldorf, Germany (poster).
Colar Zanjko, L., Meller, A., Balogh, A., Farkas, J., Chessadangkul, K., Hermans, S., Pöhlmann, T., van Roon-Mom, W., Zana, M., Dinnyés, A., “Utilizing miRNAscope™: a novel approach to compare transfection efficiency of antisense oligonucleotides across cortical cell models.” PhD Scientific days, Semmelweis University, 7-9July 2025, Budapest, Hungary (poster presentation by Laura Colar Zanjko).
Colar Zanjko, L., Balogh, A., Meller, A., Hermans, S., Pöhlmann, T., van Roon-Mom, W., Zana, M., Dinnyés, A., “Implementation of standardized in vitro toxicity assays for antisense oligonucleotide therapies for patients with ultra-rare diseases.” HUPHAR2025 International Conference of Pharmacology, Drug Research and Development, 4-6June 2025, Mátraháza, Hungary (talk given by Laura Colar Zajnko).
Dinnyés, Andras, “Challenges in Clinical Translation of Advanced Therapy Medicinal Products”, HUPHAR2025 International Conference of Pharmacology, Drug Research and Development, 4-6 June 2025, Mátraháza, Hungary.
Hermans, S., “Tailoring Antisense Oligonucleotide Manufacturing to N-of-1 Needs”, Annual Meeting of the OTS Oligonucleotide Therapeutics Society, 19-22 October 2025, Budapest, Hungary (poster).
Hermans, Seppe, “Tailoring antisense oligonucleotide manufacturing to N-of-1 needs” Dutch Antisense Therapeutic Symposium, 12-13 June 2025, Groningen.
Horvath, Rita. “Increasing Trial Readiness in AT (TREAT-AT) – an approach to developing ASO treatment for adults carrying the ‘UK’ mutation”, AT Research Conference, 25-27 June 2025, Loughborough, UK.
Kolen, I., “Personalized ASO Therapy for ALS Associated WITH KIF5A Mutations Around Exon 27: A Multi-Strategy Approach”, Annual Meeting of the OTS Oligonucleotide Therapeutics Society, 19-22 October 2025, Budapest, Hungary (poster).
Kolen, I., “Personalized ASO-therapy for ALS-associated KIF5A mutations around exon 27: a multi-strategy approach”, IZN retreat organized by interdisciplinary center for neurosciences, 13 July 2025, Schöntal, Germany (poster).
Kolen, I., “Personalized ASO-therapy for ALS-associated KIF5A mutations around exon 27: a multi-strategy approach”, Dutch Antisense Symposium, 12 June 2025, Groningen, the Netherlands (poster).
Morgan, J., “Antisense oligonucleotide-mediated exon skipping for missense variants within protein tandem-repeat domains: use case ANKRD17” 4th Dutch Antisense Therapeutics Symposium, 12-13 June 2025, University Medical Center Groningen, Groningen, the Netherlands (poster).
Morgan, J., “Antisense oligonucleotide-mediated exon skipping for missense variants within protein tandem-repeat domains: use case ANKRD17” Medical-Genetic Center South-West Netherlands (MGC) PhD Student Workshop, 3 June 2025, Düsseldorf, Germany (poster).
Risbeck, A., “Serum Proteomic Profiling in Ataxia-Telangiectasia for Biomarker Discovery”, Biomarkers & Precision Medicine, 29 September 2025 – 1 October 2025, London, UK (poster).
Schlör, L., “Covariate Model Building + PKPD Modeling of Continuous and Categorical Data”, Uppsala Pharmacometric Summer School, 11-22 August 2025, Uppsala, Sweden (poster).
Schlör, L., “Individually tailored digital-motor outcomes in real life”, MOBEX2025, 24-25 Jan 2025, Bologna, Italy.
Synofzik, M., “Bringing ASO Therapies for A-T Patients into Clinics at Scale: A Bench-to-Bedside Platform for Personalized ASO Development”, 2025 AT Clinical Research Conference, 26 June 2025, Loughborough, UK.
Synofzik, M., “A scalable platform approach for preclinical and clinical development of patient-customized ASOs for brain diseases”, MEB Science Day 2025 – “Platform Technologies: opportunities and challenges”, 19 May 2025, Utrecht, the Netherlands.
van Roon-Mom, W., “Off-Target Effects of Splice-Switching Antisense Oligonucleotides in Neuronally Differentiated Human Induced Pluripotent Stem Cells”, Annual Meeting of the OTS Oligonucleotide Therapeutics Society, 19-22 October 2025, Budapest, Hungary (poster).
van Roon-Mom, W. “Hyperindividual treatment development – a more prominent role for human derived models”, Beyond Animal Tradition symposium, 16 September 2025, Leiden, the Netherlands.
van Roon-Mom, W., “From treating many to treating 1: RNA therapeutics for brain disorders”, The Conference of the Neurological Sciences Journal, 13 May 2025, Milan, Italy.
2024
Aartsma-Rus, A., “Opportunities of antisense oligonucleotide treatments for patients with rare genetic brain and eye diseases”, Genetic Rounds Sick Kids, 17 October 2024, Toronto, Canada.
Aartsma-Rus, A., keynote lecture: “From rare to extremely rare: Applying lessons learned from Duchenne muscular dystrophy antisense oligonucleotide mediated exon skipping to developing individualized treatment”, Annual meeting of the Oligonucleotide Therapeutics Society, 6-9 October 2024, Montreal, Canada.
Aartsma-Rus, A., “Opportunities of ASO treatment for patients with ultrarare diseases”, University of San Francisco webinar series on precision medicine, 24 July 2024, online.
Aartsma-Rus, A., “From Duchenne exon skipping therapy development to individualized exon skipping for patients with rare brain or eye diseases”, Louvain University Belgium webinar series, 13 June 2024, online.
Aartsma-Rus, A., “Update on 4 years of Dutch Center for RNA Therapeutics”, Dutch Antisense Therapy Symposium, 23-24 May 2024, Rotterdam, the Netherlands.
Andrés-Santamaría, R., “Development and Validation of a Rodent Model Protocol Sensitive to Antisense Oligonucleotides Neurotoxic Effects”, LUMC neuroscience symposium, 23 October 2024, Leiden, the Netherlands (poster).
Andrés-Santamaría, R., “Development and Validation of a Rodent Model Protocol Sensitive to Antisense Oligonucleotides Neurotoxic Effects”, Annual meeting of the Oligonucleotide Therapeutics Society, 6-9 October 2024, Montreal, Canada (poster).
Andrés-Santamaría, R., “Development and Validation of a Protocol to Assess Antisense Oligonucleotides Neurotoxic Effects in a Sensitive Rodent Model”, LERU’s (League of European Research Universities) workshop “Communicating research using animals – how and why?”, 10-12 September 2024, Univerista degli Studi di Milano, Lodi, Italy.
Colar Zanjko, L., Balogh, A., Széky, B., Bódi-Jakus, M., Muenthaisong, S., Kovács-Schoblocher. D., Bánfai, K., Wiegering, A., Fehér, A., Zana, M., Schneider-Maunoury, S., Dinnyés, A., “Harnessing flow cytometry to identify discrete differences during neuronal induction of hiPSCs modeling the genetic background of Joubert syndrome.” Flow Cytometry Days workshop, 25October 2024, Semmelweis University, Budapest (poster presentation by Laura Colar Zanjko).
Hermans, Seppe, “Manufacturing Process of Antisense Oligonucleotides for N-of-1” EMA QIG LLFG on personalised medicines, 8-9 April 2024, online.
Kolen, I., Perhat, P., “1 Mutation – 1 Medicine: Tailored RNA-Therapies for ultrarare patients and diseases”, Neuro Research Day, 13 March 2024, Heidelberg, Germany (poster and presentation).
Kolen, I., “Personalized ASO-therapy for ALS-associated KIF5A mutations around exon 27: a multi-strategy approach”, Young Scientist Workshop organized by Research for Rare, 24 March 2025, München, Germany (poster).
Morgan, J., “Development of antisense oligonucleotide-mediated exon skipping therapies targeting tandem-repeat domain missense variants in ANKRD17”, LUMC neuroscience symposium, 23 October 2024, Leiden, the Netherlands (poster).
Morgan, J., “Development of antisense oligonucleotide-mediated exon skipping therapies targeting tandem-repeat domain missense variants in ANKRD17”, Annual meeting of the Oligonucleotide Therapeutics Society, 6-9 October 2024, Montreal, Canada (poster).
Morgan, J., “Individualised Exon Skipping Antisense Oligonucleotide Therapy for CHD2-Related Neurodevelopmental Disorders”, Annual meeting of the N = 1 collaborative, 5 October 2024, Montreal, Canada.
Morgan, J., “Antisense oligonucleotide-mediated exon skipping treatments for neurodevelopmental disorders associated with tandem repeat domain missense variants in ANKRD17”, Dutch Antisense Therapy Symposium, 23-24 May 2024, Rotterdam, the Netherlands (poster).
van Roon-Mom, W., “A roadmap to tailored genetic interventions in patients with neurodegenerative disorders”, ERICA ERN Research Conference, 11 December 2024, Udine, Italy.
van Roon-Mom, W., “An update on the progress of the European 1M1M network”, N1C Inaugural Symposium, 5 October 2024, Montreal, Canada.
van Roon-Mom, W., “Patient specific RNA targeting therapy for neurological disorders in the EU: Regulatory challenges”, Inaugural meeting of the UPNAT Node Symposium, 27 June 2024, London, UK.
van Roon-Mom, W., “RNA targeting therapy for neurological disorders: From treating many to treating one”, Harvard Medical School Initiative for RNA Medicine Seminar Series, 12 March 2024, online.
2023
van Roon-Mom, W. “N=1 studies and RNA therapeutics in neurodegenerative disorders”, Norwegian Society of Human Genetics, 8 November 2023, Oslo, Norway.
van Roon-Mom, W. “Splice modulating RNA targeting therapies for brain disorders; from treating many to treating one”, TIDES Europe 2023 Oligonucleotide and Peptide Therapeutics, 1 November 2023, Amsterdam, the Netherlands.