Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial

Gaucher disease type 3 is a chronic neuronopathic disorder with wide-ranging effects, including hepatosplenomegaly, anaemia, thrombocytopenia, skeletal disease and diverse neurological manifestations. Biallelic mutations in GBA1 reduce lysosomal acid β-glucosidase activity, and its substrates, gluco...

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Detalles Bibliográficos
Autores principales: Schiffmann, Raphael, Cox, Timothy M, Dedieu, Jean-François, Gaemers, Sebastiaan J M, Hennermann, Julia B, Ida, Hiroyuki, Mengel, Eugen, Minini, Pascal, Mistry, Pramod, Musholt, Petra B, Scott, David, Sharma, Jyoti, Peterschmitt, M Judith
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Clinical Trial
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9924909/
https://www.ncbi.nlm.nih.gov/pubmed/36256599
http://dx.doi.org/10.1093/brain/awac379

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9924909/
https://www.ncbi.nlm.nih.gov/pubmed/36256599
http://dx.doi.org/10.1093/brain/awac379

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