The knowledge base

The following is an interview with Sandy who lives with a severe form of LAMA2-CMD. Hello Sandy, could you present…

This week has seen the roll out of the new and updated version of the MDC1A.com website! Continuing our efforts…

Dr. Yurchenco (NJ, USA) and Dr. Rüegg (Basel, Switzerland) are collaborating with Santhera Pharmaceuticals to develop an emerging gene therapy…

The Generate Your Muscle (GYM) project was recently set up with the aim of developing and commercialising affordable stem cell…

After being diagnosed with LAMA2-CMD at 39 years old, Cindy experienced 3 seizures, the last being generalised. This prompted brain…

Dr. Dwi Kemaladewi is playing a a key role in developing a novel therapeutic approach that has opened new treatment…