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pro vyhledávání: '"Temitayo Ajayi MD"'
Autor:
Kathleen W. Wyrwich PhD, Angela Schulz MD, Miriam Nickel MD, Peter Slasor ScD, Temitayo Ajayi MD, David R. Jacoby MD, PhD, Alfried Kohlschütter MD
Publikováno v:
Journal of Inborn Errors of Metabolism and Screening, Vol 6 (2018)
Neuronal ceroid lipofuscinosis type-2 (CLN2) disease is a rare, autosomal recessive, pediatric-onset, neurodegenerative lysosomal storage disease caused by mutations in the TPP1 gene. Cerliponase alfa (Brineura®), a recombinant form of human tripept
Externí odkaz:
https://doaj.org/article/7ca9a0296a2f40ffa5f003f65090402b
