In the weekend from Friday the 15th to Sunday the 17th of November in 2019, we held the first international conference on the rare, inheritable muscular dystrophy MDC1A (Merosin Deficient Congenital muscular dystrophy type 1a). The Friday and Sunday of the conference were only accessible to experts. On Saturday the 16th patients, their parents and caregivers had the opportunity to speak to these international experts during round table sessions.
This interaction is important, because MDC1A is such a rare disease that one single physician does not exactly know how the disease progresses, nor how to measure the effects of treatments reliably, based solely on his own patients. All experts together do have this knowledge. The optimal treatment options, maintaining functions and quality of life, and the attainability of optimal care for MDC1A patients have all been topics of discussion.
The exchange of experiences was important as well. These were intense conversations that were greatly appreciated by all participants, which should lead to a higher standard of care and better treatment options for these patients. A number of experts gave presentations that were suitable for the general public. These presentations are further explained in the article below:
Written by: Prof. Dr. Bert Smeets and students of the Honours Programme Faculty of Health, Medicine and Life Sciences at Maastricht University: Marie-Julie Lemmens, Lotte van de Loo, Adnan Abdulaziz Said and Teun van Zutphen.