News
The Updated Website
This week has seen the roll out of the new and updated version of the MDC1A.com website! Continuing our efforts…
The knowledge base
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News
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Medical knowledge
A Linker Protein Gene Therapy Approach For LAMA2-CMD
Dr. Yurchenco (NJ, USA) and Dr. Rüegg (Basel, Switzerland) are collaborating with Santhera Pharmaceuticals to develop an emerging gene therapy…
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Research updates
GYM Project – A New European Collaboration
The Generate Your Muscle (GYM) project was recently set up with the aim of developing and commercialising affordable stem cell…
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Patient questions
The Experts Answer Your Questions
After being diagnosed with LAMA2-CMD at 39 years old, Cindy experienced 3 seizures, the last being generalised. This prompted brain…
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Medical knowledge
Dr. Dwi Kemaladewi On A Promising New Therapeutic Strategy For LAMA2-CMD
Dr. Dwi Kemaladewi has played a key role in developing a novel therapeutic approach, that has opened new treatment avenues…
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Medical knowledge
Prothelia: Developing A Protein Replacement Therapy For LAMA2-CMD
Steven Axon is the CEO of Prothelia, a biopharmaceutical company based in Massachusetts, USA, committed to developing a promising new…
