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LAMA2-related Muscular Dystrophy
Clinical trial pipeline · Data from ClinicalTrials.gov
See which LAMA2-related Muscular Dystrophy trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which LAMA2-related Muscular Dystrophy trials may be worth asking aboutNorth America
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The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how th…
Mutations in the LMNA gene, which codes for lamins A and C, proteins of the nuclear lamina, are responsible for a wide spectrum of pathologies, including a grou…
The goal of this observational study is to learn about the natural history and multi-organ involvement of Laminin-Alpha-2-Related Dystrophy (LAMA2-RD) in pediat…
The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future cli…
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of LAMA2-related dystrophies (LAMA2…
The goal of this natural history study is to characterize the disease course, characteristics in paediatric population of LAMA2-RD (related dystrophies) patient…
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded acces…
Mutations in the Fukutin Related Protein (FKRP) gene cause the condition Limb Girdle Muscular Dystrophy type R9 (LGMDR9) also known as LGMD2I, and the rarer con…
SEPN1 (SELENON) is a rare congenital myopathy due to mutations in the SELENON gene. MDC1A is a rare congenital muscle dystrophy due to mutations in the LAMA2 ge…