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  3. Natural History of Duchenne Muscular Dystrophy: A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years old.

Natural History of Duchenne Muscular Dystrophy: A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years old.

Datos básicos

Código:
GNT-014-MDYF
Protocolo:
GNT-014-MDYF
EUDRACT:
NCT:
Centro:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Dotación:
Año de incio:
2024
Año de finalización:
PI BIOMÉDICA

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Participantes

Grupos

Financiadores - Promotores

GENETHON

Resultados del Ensayo Clínico

Asymptomatic HyperCKemia in the Pediatric Population A Prospective Study Utilizing Next-Generation Sequencing and Ancillary Tests

Marti, Pilar; (...); Vilchez, Juan Jesus

Article. 10.1212/WNL.0000000000210116. 2025


Clinical features, mutation spectrum and factors related to reaching molecular diagnosis in a cohort of patients with distal myopathies.

Muelas, Nuria; (...); Vilchez, Juan J

Article. 10.1007/s00415-024-12821-3. 2025


Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model

Poyatos-Garcia, Javier; (...); Vilchez, Juan J

Article. 10.1186/s13395-024-00353-3. 2024

  • Open Access.

Muscle MRI-phenotyping of patients with likely pathogenic anoctamin 5 variants

Poulsen, N.; (...); Vissing, J.

Meeting Abstract. 10.1016/j.nmd.2024.07.187. 2024

  • Open Access.

Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy

Vucinic, D.; (...); Muelas, N.

Meeting Abstract. 10.1016/j.nmd.2024.07.289. 2024

  • Open Access.

Spanish Pompe Registry: overview based on the 130 patients included

Marin, R. Martinez; (...); Manera, J. Diaz

Meeting Abstract. 10.1016/j.nmd.2024.07.732. 2024

  • Open Access.

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