We are delighted to invite all those affected by myotubular and centronuclear myopathies their families, doctors and carers to our family conference. As in 2016, the conference will be hosted jointly by the Myotubular Trust and ZNM – Zusammen Stark! (CNM – Together Strong!)
Friday, July 13th
Informal get-together as and when people arrive and settle in.
Saturday, July 14th, 9 am – 6 pm: conference programme
The Conference, chaired by Professor Francesco Muntoni will be presented in both German and English. Scientific presentations will include recent developments on potential treatments for all centronuclear myopathies, such as gene therapy, DNM2 down-regulation and drug repurposing, among others.
Head of the Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, and Great Ormond Street Hospital
“Overview of progress made, and lessons learnt in other neuromuscular diseases”
“Gene therapy development”
Department of Translational Medicine and Neurogenetics, I.G.B.M.C, France
” DNM2 downregulation as a potential treatment for a number of centronuclear myopathies”
Professor and Consultant in Paediatric Neurology, Children’s Neuroscience Centre, St Thomas’ Hospital, London
“Other CNM genes – our latest understanding – RYR1 and Titin related myopathies”
Clinician, Hospital for Sick Children, Toronto, and Assistant Professor, Departments of Paediatrics and Molecular Genetics, University of Toronto
“Drug Repurposing for myotubular myopathy”
Professor of Respiratory & Sleep Medicine, National Heart & Lung Institute and Respiratory Consultant at Royal Brompton & Harefield Hospital, London
“Latest international standards of ventilatory choices in NMD”
Senior Investigator, Neurogenetics Branch, Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Health (NIH), Maryland
“Study design and burden on families in clinical trials for ultra rare diseases”
Saturday 14th 2pm-6pm & Sunday, July 15th, 9am–12 noon
Presentations from industry
• Genetic background, options and counselling
• Living with long term ventilation and latest in airway clearance standards for NMD
• Psychological support for the whole family
• Wheelchairs and postural support advice
• Travelling and holidays
• Independent living
• Fundraising for research
• Nutrition and blended foods
In the afternoon we are planning a series of workshops on topics such as ventilation, physiotherapy, nutrition, independent living, holidaying and wheelchair services. If you have any specific wishes or ideas, do feel free to contact us with your suggestions – firstname.lastname@example.org
Sunday, July 15th, 9 am – 12 noon: conference programme and closing
Registration for the conference
We are very grateful for the many donations big and small that we have received in order to organise this conference. An overview on our donors is at the end of this page.
Because of these donations, attendance at the conference, including lunch and dinner, are free for those affected by myotubular and centronuclear myopathy, their families and carers. However, you will be responsible for your own accommodation costs.
Other guests are welcome to attend the conference for the net cost of £100 per person per day including lunch, dinner and coffee breaks. Accommodation is at your own expense.
Between now and the conference, the organising team will be working hard at fundraising to pay for this event. If you can help in any way or have any ideas, we would be immensely grateful and would love to hear from you.
There will be a full children’s programme for different age ranges, designed, managed and staffed by professional and experienced child care providers.
As mentioned above, the family conference will be simultaneously translated from English to German and vice versa. If you do not speak English or German, but still would like to attend the conference, please contact Jennifer Bilbao
Members of our organizations also speak Croatian, Turkish, Spanish, Dutch as mother tongue and are very happy to help you in case you have any detailed questions.
Without the many donations it would not have been possible to organise this conference. Here is the place where we ant to say Thank you!
Foundation Kindness for Kids
Kindness for Kids was founded in 2003 with the goal to improve the situation of children with rare diseases. Kindness for Kids’ approach is two-fold: at a social as well as at a scientific level.
Socially, the foundation organizes and funds holiday camps with medical assistance for affected children and vacations assisted by therapists for the whole family.
Scientifically, the foundation commits itself in the research funding field with a focus on health care.