Announced details
Treatment development for LAMA2-related congenital muscular dystrophy (LAMA2-RD)
Demonstrated significant drug efficacy and effectiveness in disease model mice, and confirmed safety and expression of the target gene LAMA1 in a non-human primate study (quantitative data: Not disclosed)
In this announcement the company addresses none of Contract Value / Offering price / Impact on Business Performance.
2026 LAMA2 Conference
| Item | Details |
|---|---|
| Names of Products / Services Covered | MDL-101, CRISPR-GNDM® |
| Launch date | 29 March 2026 (scheduled; date and time of the oral presentation at the conference) |
| Applicable area | Treatment development for LAMA2-related congenital muscular dystrophy (LAMA2-RD) |
| Quantitative Effects | Demonstrated significant drug efficacy and effectiveness in disease model mice, and confirmed safety and expression of the target gene LAMA1 in a non-human primate study (quantitative data: Not disclosed) |
| Source | 2026 LAMA2 Conference |
Items this document does not state (13)
These are items the company does not state in this document. We do not leave them blank, nor fill them in from other sources or by inference.
The reasons the company gives
The company's own explanation, not something inferred from the numbers.To present at an international platform aimed at sharing research findings, facilitating dialogue with experts and the patient community, and accelerating the development of care and treatments for LAMA2-related congenital muscular dystrophy (LAMA2-RD).