Results 111 to 120 of about 690 (122)

EM.P.1.04 Treatment approaches in laminin-α2-deficient congenital muscular dystrophy (MDC1A)

Neuromuscular Disorders, 2009
S. Meinen, S. Lin, M.A. Rüegg
openaire   +1 more source

Widening the spectrum of LAMA 2 congenital muscular dystrophy (MDC1A): cobblestone malformation

Arquivos De Neuro-Psiquiatria, 2022
Thiago Rezende, Paula Maria Preto Mimura, Fernanda Veloso Pereira   +2 more
exaly  

P289 – 2042 Merosin-deficient congenital muscular dystrophy type 1A (MDC1A): a case report

European Journal of Paediatric Neurology, 2013
C Graf von Kalckreuth, N Deconinck, J Goedseels, A Neuman, E Chevalier, H Kadhim, PQ Lê   +6 more
openaire   +1 more source

C.P.2.13 Widening the LAMA2 mutation spectrum in MDC1A patients: New mutations including gross deletions

Neuromuscular Disorders, 2007
J. Oliveira, I. Soares-Silva, P. Jorge, E. Vieira, M. Oliveira, R. Santos   +5 more
openaire   +1 more source

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Journal of Neuromuscular Diseases, 2015
Mahasweta Girgenrath, Anthony Accorsi, Younghwa Rhee   +2 more
exaly  

Development of intein-based tripe AAV gene therapy for MDC1A disease: expanding AAV capacity to treat monogenic disease

During the development of this project, we have focused on expanding the adeno-virus associate (AAV) delivery tool capacity to potentially treat any monogenic disease. Several AAV-based therapies have obtained marketing authorization in recent years. Nonetheless, the potential use of AAVs is constrained by their limited gene packaging capacity, which ...
openaire   +1 more source

G.P.211: Combination therapy with Glatiramer acetate and FTS improves strength and fibrosis in the dy2J/dy2Jmouse model of MDC1A

Neuromuscular Disorders, 2014
M Rabie, S Mitrani-Rosenbaum
exaly  

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Frontiers in Aging Neuroscience, 2014
Madeleine Durbeej, Azra Alajbegovic, Johan Holmberg   +2 more
exaly  

Severe MDC1A Congenital Muscular Dystrophy Due to a Splicing Mutation in the LAMA2 Gene Resulting in Exon Skipping and Significant Decrease of mRNA Level

Genetic Testing and Molecular Biomarkers, 2007
Nacim Louhichi, Pascale Richard, Faouzi Baklouti   +2 more
exaly  

Laminin alpha2 negative congenital muscular dystrophy (MDC1A) presenting with a mild phenotype

2003
PRANDINI P, BERARDINELLI A, FANIN, MARINA, ANGELINI, CORRADO, PEGORARO, ELENA   +4 more
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