European Family Conference 2018 – July 14 & 15, London

#TogetherEvenStronger2018

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!)

All participants / alle Teilnehmer
All participants of the Family Conference 2016 in Frankfurt

Outline programme

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.

 Francesco Muntoni
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”

Ana Buj-Bello
Genethon, France
“Gene therapy development”

Belinda Cowling
Department of Translational Medicine and Neurogenetics, I.G.B.M.C, France
” DNM2 downregulation as a potential treatment for a number of centronuclear myopathies”

Heinz Jungbluth
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”

James Dowling
Clinician, Hospital for Sick Children, Toronto, and Assistant Professor, Departments of Paediatrics and Molecular Genetics, University of Toronto

“Drug Repurposing for myotubular myopathy”

Anita Simonds
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”

Carsten Bonnemann
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

Audentes Therapeutics: an update on the ASPIRO gene therapy clinical trial
Dynacure: an update on DNM2 antisense oglionucleotide clinical trial programme development

Workshops including:
• 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 – contact@myotubulartrust.org

Sunday, July 15th, 9 am – 12 noon: conference programme and closing

Further workshops

Venue

Holiday Inn, Bloomsbury Coram Street, London WC1N 1HT – this hotel is a few streets away from the world renowned Great Ormond Street Hospital for Children

Registration for the conference

Registration form is available here.

Costs

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.

Spiel draußen / playing outside
Children programme during the Family Conference 2016 in Frankfurt

Children’s programme

There will be a full children’s programme for different age ranges, designed, managed and staffed by professional and experienced child care providers.

Conference languages

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.

 

Please watch the Myotubular Trust and ZNM – Zusammen Stark e.V. (CNM – Together Strong!) websites for updates and further information, including the full agenda.

 

Thank you

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.