Spanish Natural History Study for LAMA2 Muscular Dystrophy
Recruiting
Observational Study
LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)
Merosin Deficient CMD (Full or Partial)
Merosin Deficient Congenital Muscular Dystrophy
Muscular Dystrophies
Cohort Studies
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 0 Minutes – 100
- Sex
- Any
- Study type
- Observational
- Participants needed
- 100 (estimated)
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute · Other
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About This Trial
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of LAMA2-related dystrophies (LAMA2-RD) in the pediatric population. The study aims to establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* All patients with compatible clinical presentation and identification of 2 pathogenic variants in LAMA2, or muscle biopsy with decreased laminin alpha2 protein and at least one pathogenic variant
* Signed informed consent by the Legal Authority Responsible and/or assent by the…
Contacts