Annex 1
Overview of meetings where the Network was/will be presented or mentioned
Presentations focusing on the Action/discussing the Action (for at least 5 minutes)
Presenter: Annemieke Aartsma-Rus
Date: 15-17 April 2013
Location: Dublin, Ireland
Meeting: International conference of the International Rare Disease Research Consortium (IRDiRC)
Audience type: Scientists, clinicians and patient representatives
Audience size: ~ 300
Title talk: Antisense-mediated exon skipping – Applying lessons learnt from Duchenne muscular dystrophy to other rare diseases
Presenter: Annemieke Aartsma-Rus
Date: 17 May 2013
Location: Salt Lake City, Utah, USA
Meeting: ASGCT satellite meeting of the Gene and Cell Therapy Consortium
Audience type: Scientists and clinicians
Audience size: ~100
Title talk: Networking – the TREAT-NMD paradigm
Presenter: Annemieke Aartsma-Rus
Date: 13 Sept 2013
Location: Leiden, the Netherlands
Meeting: Gene therapy workshop of the Dutch Organisation of Bone Marrow Transplantation
Audience type: scientists and clinicians
Audience size: ~150
Title talk: Opportunities of antisense-mediated exon skipping
Presenter: Virginia Arechevala-Gomeza
Date: 16-19 October 2013
Location: San Sebastian, Spain
Meeting: International Myotonic Dystrophy Consortium
Audience type: Scientists and Clinicians
Audience size: ~200-300
Title poster: Networking towards clinical implementation of antisense-mediated exon skipping
Presenter: Annemieke Aartsma-Rus
Date: 23-26 October 2013
Location: Turin, Italy
Meeting: Rare Disease symposium of the Italian Pharmacological Society
Audience type: Pharmacologists
Audience size: ~100
Title talk: Antisense-mediated exon skipping – a therapeutic approach with potential for DMD and other rare diseases
Presenter: Annemieke Aartsma-Rus
Date: 29 October – 1 November
Location: Newcastle, UK
Meeting: TREAT-NMD symposium
Audience type: Scientists, clinicians, industry representatives, patient representatives
Audience size: ~ 300-400
Title talk: Joint research and regulatory interaction
Presenter: Annemieke Aartsma-Rus
Date: 17 December 2013
Location: Bonn, Germany
Lecture: Leiter des Schwerpunkts Klinische Neuroonkolgie Lecture
Audience type: Clinicians and scientists
Audience size: ~ 100
Title talk: Antisense-mediated exon skipping for Duchenne muscular dystrophy and other neurological disorders
Presenter: Annemieke Aartsma-Rus
Date: 21-23 February 2014
Location: Rome, Italy
Meeting: Italian Duchenne Parent Project Onlus
Audience type: parents, patients, scientists, clinicians, industry representatives
Audience size: ~400
Title talk: Preclinical exon skipping studies
Presenter: Annemieke Aartsma-Rus
Date: 24-26 February 2014
Location: Heidelberg, Germany
Meeting: RD-Connect, NEUROMICS and EURENOMICS joint progress meeting
Audience type: scientists, clinicians
Audience size: ~500
Title talk: Antisense-mediated exon skipping for DMD and other rare diseases
Presenter: Annemieke Aartsma-Rus
Date: 13-14 March 2014
Location: Lunteren, the Netherlands
Meeting: Annual meeting of the Dutch Society of Gene and Cell Therapy
Audience type: clinicians, scientists, industry representatives
Audience size: ~ 100
Title talk: Antisense-mediated exon skipping: An approach with therapeutic potential for DMD and other diseases
Presenter: Annemieke Aartsma-Rus
Date: 27-28 March 2014
Location: Porto, Portugal
Meeting: Genetics Conference
Audience type: geneticists
Audience size: ~200
Title talk: Antisense-mediated exon skipping for Duchenne muscular dystrophy – clinical trials and beyond
Presentations mentioning the Network
The Network was also mentioned during the Oligonucleotide Therapeutics Society meeting (6-9 October 2013, Naples Italy) by Mike Gait when he chaired a session. He also mentioned the opportunity for people to join the Action (resulting in Denmark signing up).
Presenter: Francesco Muntoni
Date: April 21-24 2013
Location: Washington DC, US
Meeting: Muscular Dystrophy Association Scientific Conference
Audience type: scientists, clinicians and patient representatives
Audience size: ~500
Title talk: What we have learned from clinical trials to date
Presenter: Annemieke Aartsma-Rus
Date: April 24 2013
Location: Washington DC, US
Lecture at Children’s National Medical Center
Audience type: scientists and clinicians
Audience size: 100
Title presentation: the development of antisense-mediated exon skipping for DMD
Presenter: Willeke van Roon-Mom
Date: 11-13 June 2013
Location: Paris, France
Meeting: SPATAX
Audience type: scientists, clinicians and patients
Audience size: 200
Title presentation: Using antisense oligonucleotides to reduce or modify the disease causing proteins in autosomal dominant spinocerebellar ataxis
Presenter: Annemieke Aartsma-Rus
Date: August 22 2013
Location: Tanjin University, China
Lecture at Tanjin University
Audience type: scientists
Audience size: 20
Title presentation: Antisense-mediated exon skipping for DMD
Presenter: Lourdes Desviat
Date: September 3-6 2013
Location: Barcelona, Spain
Meeting: International Congress of Inborn Errors of Metabolism
Audience type: Scientists and Clinicians
Audience size: ~500
Title poster presentation: In vivo effect of splice modulating antisense oligonucleotides: generation of hyperphenylalaninemia mice as model
Presenter: Zaida Koeks
Date: 7 September 2013
Location: Veldhoven, the Netherlands
Meeting: Muscle Congress of the Dutch Neuromuscular Patient Organisation
Audience type: Patients and parents
Audience size: ~80
Title talk: Overview of medication trials in Duchenne muscular dystrophy
Presenter: Francesco Muntoni
Date: 30-31 October 2013
Location: Newcastle UK
Meeting: TREAT-NMD symposium
Audience type: Scientists, clinicians, industry representatives, patient representatives
Audience size: ~ 300-400
Title talk: Emerging therapies for SMA
Presenter: Francesco Muntoni
Date: 1-2 November 2013
Location: Athens, Greece
Meeting: Mediterranean Society of Myology
Audience type: Scientists, clinicians
Audience size: ~ 200
Title talk: Genetic therapies in muscular dystrophies
Presenter: Annemieke Aartsma-Rus
Date: November 7 2013
Location: Helsinki, Finland
Meeting: Annual meeting of the Finish Society of Pediatric Neurology in memory of Martha Donner
Audience type: Clinicians
Audience size: ~ 200
Title talk: Preclinical development of antisense-mediated exon skipping for DMD and other neurologic diseases
Presenter: Annemieke Aartsma-Rus
Date: November 20-22 2013
Location: Cardiff, UK
Meeting: Cardiovascular genetics symposium
Audience type: Cardiologists, clinical geneticists
Audience size: ~ 400-500
Title talk: Antisense-mediated exon skipping for Duchenne muscular dystrophy
Presenter: Annemieke Aartsma-Rus
Date: January 11, 2014
Location: Chennai, India
Meeting: Myocon
Audience type: clinicians, researchers, parents
Audience size: ~ 200-300
Title talk: Future directions of antisense-mediated exon skipping for Duchenne muscular dystrophy
Presenter: Lourdes Desviat
Date: May 20-24 2014
Location: Washington DC USA
Meeting: American Society of Gene and Cell Therapy
Audience type: Scientists and Clinicians
Audience size: ~1000
Title oral or poster presentation: A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in mouse
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