Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Vivian Kessler"'
Autor:
Andrew E. Mulberg, Christina Bucci-Rechtweg, Joseph Giuliano, David Jacoby, Franklin K. Johnson, Qing Liu, Deborah Marsden, Scott McGoohan, Robert Nelson, Nita Patel, Klaus Romero, Vikram Sinha, Sheela Sitaraman, John Spaltro, Vivian Kessler
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-10 (2019)
Abstract Rare or orphan diseases often are inherited and overwhelmingly affect children. Many of these diseases have no treatments, are incurable, and have a devastating impact on patients and their families. Regulatory standards for drug approval fo
Externí odkaz:
https://doaj.org/article/66e8a255f71a491c85332834df6a087b
Autor:
Andrew E. Mulberg, Alan L. Shields, Vivian Kessler, Nina Skuban, Jay A. Barth, Fiona Taylor, Roger E. Lamoureux
Publikováno v:
Quality of Life Research
Purpose Fabry disease is a rare multisystemic disorder caused by functional deficiency of the lysosomal enzyme alpha-galactosidase A. Gastrointestinal (GI) signs and symptoms are among the earliest clinical manifestations in patients with Fabry disea
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::dbbb77d88c3b8ff9df1ed40f9a8e7799
https://doi.org/10.1007/s11136-021-02847-9
https://doi.org/10.1007/s11136-021-02847-9