News
A look back on a conference to shape LAMA2 clinical care guidelines
We are delighted to reflect on the success of the ENMC conference, initiated by Voor Sara and Cure CMD. Together,…
Research updates
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News
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Research updates
Spain’s ongoing LAMA2-CMD natural history study
In 2022, a natural history study was initiated with LAMA2-CMD patients at Barcelona’s Vall d’Hebron Institute of Research. Natural history…
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News
One step closer: FDA grants Rare Pediatric Disease designation to Modalis Therapeutics’ MDL-101 gene therapy
Modalis Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to…
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News
Workshop to establish subtype-specific care recommendations for LAMA2-CMD
We are thrilled to announce a new clinical workshop focused on establishing subtype-specific care recommendations for LAMA2-RD, and using this experience as a prototype to develop recommendations for…
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News
Dr. Voermans secures funding for extension of LAMA2-CMD natural history study
In 2022, Dr. Nicol Voermans and her team at Radboud UMC, Netherlands, launched the LASTSTRONG natural history study. With new…
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Research updates
Modalis Therapeutics advances a novel gene therapy for LAMA2-CMD
Modalis Therapeutics has made significant strides in developing a gene therapy technology aimed at treating LAMA2-congenital muscular dystrophy (LAMA2-CMD). Earlier…
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