Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Niek P. vanTil"'
Autor:
Marianna Bugiani, Truus E. M. Abbink, Arthur W. D. Edridge, Lia van derHoek, Anne E. J. Hillen, Niek P. vanTil, Gino V. Hu‐A‐Ng, Marjolein Breur, Karen Aiach, Philippe Drevot, Michaël Hocquemiller, Ralph Laufer, Frits A. Wijburg, Marjo S. van derKnaap
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 10, Iss 6, Pp 904-917 (2023)
Abstract Objective Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease‐modifying therapy is available. The AAVance gene therapy trial
Externí odkaz:
https://doaj.org/article/7b120ba2fed845d38eb844ce2a9e52f1
Autor:
Maud Plantinga, Niek P. vanTil, Stefan Nierkens, Colin de Haar, Rob C. Hoeben, Jaap Jan Boelens, Manuel A F V Gonçalves, Lea Knežević
Publikováno v:
Molecular Therapy. 24:S160
Hematopoietic stem cell transplantation is the standard of care for pediatric acute myeloid leukemia (AML) patients, but relapse still occurs in approximately 40% of AML patients. Hence there is an unmet need to improve disease control with fewer adv
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::8794a7816a72feb602b1e976c6fe6141
https://doi.org/10.1016/s1525-0016(16)33213-0
https://doi.org/10.1016/s1525-0016(16)33213-0