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!).

IMPORTANT: Please note  discounted accommodation rate has expired on 15th June.  See ‘Venue’ details below for further information.

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.

9.00  Registration and coffee

9.30  Opening and welcome by Myotubular Trust and ZNM Zusammen Stark!

CONFERENCE PROGRAMME

“Overview of progress made, and lessons learnt in other neuromuscular diseases”
Francesco Muntoni
Head of the Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, and Great Ormond Street Hospital

“Genes, and the genetic background of centronuclear myopathy”
Johann Böhm
Department of Translational Medicine and Neurogenetics, IGBMC, France

“Progress in gene therapy development for myotubular myopathy”
Ana Buj-Bello
Genethon, France

“DNM2 downregulation as a potential treatment for a number of centronuclear myopathies”
Belinda Cowling
Department of Translational Medicine and Neurogenetics, IGBMC, France

“RYR1 and TTN – the challenge of giant genes implicated in centronuclear myopathy”
Mathias Gautel
Chair of Molecular Cardiology, and Head of School of Basic and Medical Biosciences, King’s College, London, UK and
Heinz Jungbluth
Professor and Consultant in Paediatric Neurology, Children’s Neuroscience Centre, St Thomas’ Hospital,

“Drug Repurposing for myotubular myopathy”
James Dowling
Clinician, Hospital for Sick Children, Toronto, and Assistant Professor, Departments of Paediatrics and Molecular Genetics, University of Toronto

“Latest international standards of ventilatory choices in NMD”
Anita Simonds
Professor of Respiratory & Sleep Medicine, National Heart & Lung Institute and Respiratory Consultant at Royal Brompton & Harefield Hospital, London

“Study design and burden on families in clinical trials for ultra rare diseases”
Carsten Bonnemann
Senior Investigator, Neurogenetics Branch, Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Health (NIH), Maryland

13.00 – 14.30    Lunch

 

14.30 – 18.00   Afternoon sessions

Workshops Programme:

“An updated overview on the genes implicated, and genetic testing for myotubular and centronuclear myopathy, including reproductive advice” 
Meriel McEntagart
Consultant Geneticist, St George’s Hospital, London, UK

“Update on respiratory care: what’s new and what works” 
Anita Simonds
Consultant in Respiratory Medicine, Royal Brompton Hospital, London, UK

“Respiratory management – your questions answered” 
Dr Michelle Chatwin
Clinical Specialist Physiotherapist, Department of Sleep and Breathing, Royal Brompton Hospital, London, UK

“Tracheostomies – dispelling the myths:putting the person first” 
Dr Debbie Field
Specialist Outreach Nurse, Complex Ventilation, Tracheostomy and Weaning, Royal Brompton Hospital, London, UK

“Fundraising – a brainstorm” 
Open forum
Your ideas, suggestions and recommendations greatly appreciated

“An update on the ASPIRO gene therapy programme for x-linked myotubular myopathy” 
Audentes Therapeutics
San Francisco, USA

“Support in times of bereavement” 
Heather Tilley
Family Support & Bereavement Counsellor, BACP, UK

18.30 – 20.30   Buffet dinner – all invited


Sunday, July 15th:

09.00 – 12.00   Sunday sessions

Workshops Programme:

“Independent living” 
Shaun Marsh and Zak Hughes
Adult patients from our community, UK

Supporting parents and carers with the needs and experiences of sibling children” 
Heather Tilley
Family Support & Bereavement Counsellor, BACP, UK

“Goals beyond grass – an introduction to power chair football” 
Wheelchair Football Associations Representatives from UK and Switzerland

“DNM2 mutations – explanation, prognosis and therapies” 
Johann Böhm
Department of Translational Medicine and Neurogenetics, IGBMC, France

“Holidays and travel planning” 
Open forum
A family workshop to share ideas and experiences

“An update on the pre-clinical work on Dynamin 2 downregulation for myotubular and centronuclear myopathies” 
Dynacure
Strasbourg, France

“Blended food – supporting parents and patients who choose a blended diet for gastrostomy tube feeding” 
Suzanne Brown 
RSCN, Children’s Hospice Southwest, Devon, UK

12.00pm           Closing


 

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

To avail of the discounted rate for the event, please use group code KUH. Rooms including breakfast cost £168 per night plus £10 supplement per person for multiple room occupancy. You can book online or per phone + 44 207 923 6651. The cut off date for booking rooms is 15th June.

Here you can find details about other wheelchair accessible hotels that are within walking distance to the Conference.

There is paid parking beneath the hotel (£28 per day).

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 and teenager programme

This is very much a family event and you are welcome to reserve a place for your children in our under 16s workshop, being held in a room right next door to the conference.

The Children’s Workshop will be expertly organised and managed by Alison Dyke, an experienced and qualified Nursery Owner and Manager, who is also RGA Nurse trained. Alison will be accompanied by experienced childcare volunteers, and nurses.

The programmes will include toys, fun and games, balloons and magic, petting animals and critters, cooking, art, karaoke and open mic, music, books, films, games, x-box and PS4 multi gaming, gardening and more!

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.

 

 

 

 

 

 

The Boshier-Hinton Foundation exists to improve quality of life for people with disabilities, learning difficulties or sensory impairement and their families, by the awarding of grants.

 

 

Audentes Therapeutics, Inc. (PRNewsFoto/Audentes Therapeutics, Inc.)

Breas provide innovative solutions that enable people with chronic respiratory conditions to live life to the fullest.

 

Our thanks also to Cynthia Dickenson Charitable Trust.