Dutch non-profit foundation Voor Sara promotes research into the causes and treatments of the rare muscle disease MDC1A / Lama2 CMD. For this website we work together with the Spanish patient organisation ImpulsaT, the pharmaceutical company Prothelia and various scientists and doctors. This website was made possible thanks to the financial support of Prothelia and ImpulsaT.
Dutch non-profit foundation Voor Sara promotes research into the causes and treatments of rare muscle diseases in general and specifically MDC1A / Lama2 CMD. The foundation seeks to achieve its goals by bringing attention to muscle diseases, raising funds, and giving all those involved the chance to come together, exchange information and drive innovation.
Foundation Voor Sara was established at the end of 2016 and already made research into MDC1A possible at Maastricht University. Professor Bert Smeets works on stem cell therapy for muscle diseases. Together with the university the foundation brings leading medical researchers and doctors together to make important steps into a therapy for MDC1A. The foundation, together with Maastricht University and the Radboud hospital in Nijmegen, has also set up a natural history study for 12 Dutch-speaking patients.
In America, Spain, the Netherlands and in so many more places, people are working hard for the Lama2 community. Everyone works towards the same goal and we are very grateful and proud that we have noticed in recent months that everyone also enjoys working together. For this website we work together with the Spanish patient organisation ImpulsaT, the pharmaceutical company Prothelia and various scientists and doctors. This website was made possible thanks to the financial support Prothelia and ImpulsaT.
By highlighting all this commitment on one website, we make it clear how much is happening in this field. We believe this can help raise new funds, keep scientists engaged, inspire new researchers and keep the flywheel going. We also hope to find new partners in for example Asia and the Middle East. And just as important: to keep you well informed.
In this way, we hope to create an international platform where all important information can be centralized and found by everyone involved in the struggle for treatment. We already have good contacts with many enthusiastic people, but probably don’t know everyone yet. If we have missed a patient organisation, an investigation or something else important, please let us know!
We hope to be able to form an international community together, because together we are stronger and can achieve more.
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. MDC1A.com 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.