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…
The knowledge base
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Medical knowledge
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Research updates
Ph.D. Karlijn Bouman: understanding the progression of LAMA2-CMD
Drs. Karlijn Bouman (PhD Candidate) is part of a team of Dutch doctors and researchers that are carrying out a…
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Research updates
Dr. Carsten Bönnemann on LAMA2-CMD clinical trial readiness
Dr. Carsten Bönnemann and Dr. Reghan Foley are running a natural history study for infants aged 0-5 years of age.…
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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…
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Research updates
Dr. Avencia Sanchez-Mejías’ ex-vivo therapeutic approach to LAMA2-CMD
Dr. Avencia Sanchez-Mejías has been investigating how haematopoietic stem cells from LAMA2-CMD patients’ bone marrow, can be genetically corrected to…
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Medical knowledge
LAMA2-CMD, MDC1A and Limb Girdle Dystrophy: What’s the difference?
Limb Girdle Dystrophy, MDC1A and LAMA2-CMD. Why are there three different names to describe the one and same disease? In…
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