Join the conference

Conference

Saturday 20 November

The speakers

10.55 - 11.00

Opening

By K. Huisman

Opening with B. Verbrugge, H. Smeets, D. Linden and Saturday chair K. Huisman

11.00 - 11.30

The clinical spectrum of MDC1A

By C. Bönneman

C. Bönneman, M.D. works at the National Institute of Health in the USA, where he researches the underlying molecular mechanisms of muscle diseases.

11.30 - 12.00

Clinical management of MDC1A

By H. Sawnani

H. Sawnani, M.D. is a pediatric pulmonologist at the Cincinnati Children’s Hospital in the USA. His interest lies with pulmonary care for patients with muscular dystrophy.

12.00 - 12.30

Genotype-phenotype correlations in MDC1A and CMD

By A. Sarkozy

Honorary Senior Lecturer and Consultant in Neuromuscular Diseases at the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health and Great Ormond Street Hospital for Children, London, UK

12.30 - 13.30

Lunch buffet

By The organisation
13.30 - 14.00

Therapeutic strategies for MDC1A

By M. Durbeej

Head of the Department of Experimental Medical Science and Professor of Muscle Biology at Lund University, Sweden

14.00 - 14.30

Gene therapy for MDC1A

By D. Kemaladewi

D. Kemaladewi is an Assistant Professor at the University of Pittsburgh, USA. Her research focusses on underlying molecular mechanisms of muscular dystrophies.

14.30 - 15.00

Stem cell therapy in congenital MD

By M. Sampaolesi

Head of the Laboratory of Translational Cardiomyology in Stem Cell and Developmental Biology and Professor at the University of Leuven, Belgium

15.30 - 17.30

Round tables: Meet the experts

Meet the experts for patients, caregivers and professionals

17.30 - 19.30

Walking Dinner Buffet

By The organisation

Frequently asked questions

MDC1A is a hereditary muscle disorder often noticed at birth or within the first few months of life. It is a type congenital muscular dystrophy (CMD) and has an incidence of 1 to 4 people in every 100.000. MDC1A is caused by a defect in the LAMA2 gene which is responsible for the production of laminin α2 protein (previously called merosin). MDC1A is sometimes referred to as LAMA2-related muscular dystrophy.

Saturday November 16, we will organize in Maastricht a one-day conference on the road to therapy for MDC1A. During that conference over 25 world-renown experts in the field of MDC1A or congenital muscular dystrophies will be around.

The programme consists of 3 parts. First, we will start with a series of 6 lectures, half of them on clinical issues and half of them on therapy development. The first lecture gives an overview of the theme (clinical spectrum, therapy) and the other lectures will be more in-depth by researchers sharing their recent exciting findings. Topics are based on input from the patient organization “Voor Sara”. After the first 3 lectures there will be lunch.

Following the last lecture we have 3 round-table sessions, allowing you to learn about the topics you are most interested in and discuss these with world-experts in the field. Each table has a theme and 1 or 2 experts allocated to them. For the Dutch speaking participants, we also have 2 round tables explaining the presentations in Dutch and 2 tables explaining the clinical and therapeutic research done in the Netherlands. Please select upon registration which table you would like to join in which session by indicating the number of that session. You can choose 3, one table per session.

After the round table sessions, a walking dinner will be served, allowing you to interact on a one-to-one basis with all other participants. But also to exchange experiences and have fun. We hope that by the end of the day, everybody has achieved what they wanted and has established contacts and a network to fulfil his or her own interests and needs in the future. We intend to make these contacts sustainable by organizing this meeting on a regular basis.

The conference is initiated by Maastricht University and Stichting Voor Sara. This congress will address an overview of the pathophysiology and the challenges in treating congenital muscular dystrophies and in particular MDC1A.
Stichting Voor Sara is a Dutch non-profit foundation which promotes research into the causes and treatments of rare muscle diseases in general and specifically MDC1A. The foundation seeks to achieve its goals by raising funds, bringing attention to muscle diseases and giving the chance to all those involved to come together and exchange information. More information about the foundation can be found at www.voorsara.nl.
The main goal is to create an overview of the pathophysiology and the challenges in treating congenital muscular dystrophies, and in particular MDC1A. International and local experts can share their findings on the latest innovative therapeutic treatments, current practices and more. The conference will further be a platform for all those involved in congenital muscle dystrophies, as patient foundations and members of the public can join certain activities in the aim of raising more awareness. A voice will be given to those affected by muscular dystrophies, so that we can explore what can be done better and what else can be focused on.
The second day of the event, on Saturday the 16th of November, is open for anyone who wants to participate. Doctors, researchers, physiotherapists, occupational therapists and patients who suffer from MDC1A are invited. Tickets for this public part of the conference can be purchased on this website, via the following link.
Tickets for this public part of the conference can be purchased on the website below these questions.
You can get a full refund if you cancel your ticket 30 days before the event. If you cancel after that you will not be refunded.
By making a donation you automatically support the conference and you will also be supporting research to be done into MDC1A.
You can also make a donation if you have a business or if you want to be a sponsor. All donations are tax deductible.
Foundation Voor Sara works closely with a number of independent medical advisors who evaluate the researches. Also the Princess Beatrix Muscle Fund and the Dutch Muscle Disease Patients Association advise us. This last party has written a letter of support for Professor Smeets’ research into muscle stem cell therapy. The international patients organisation also supports the research. Part of the research is funded by the Princess Beatrix Muscle Fund. All parties reviewed the research promising, ambitious and see it as very valuable in the search for a method for maintaining muscle mass.
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