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…
Medical knowledge
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Medical knowledge
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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…
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Medical knowledge
Join the Congenital Muscle Disease International Registry
The Congenital Muscle Disease International Registry (CMDIR) aims to advance neuromuscular research by collecting vital data and by growing advocacy…
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Research updates
Ph.D. Karlijn Bouman: understanding the progression of MDC1A
Drs. Karlijn Bouman (PhD Candidate) is part of a team of Dutch doctors and researchers that are carrying out a…
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Medical knowledge
Get to know the work of respiratory therapist Ruud van der Wel
As a respiratory therapist and an innovator, Ruud van der Wel has been able to develop amazing tools that can…
