Zobrazeno 1 - 10
of 91
pro vyhledávání: '"Andrew E, Mulberg"'
Autor:
Nathan Denton, Andrew E. Mulberg, Monique Molloy, Samantha Charleston, David C. Fajgenbaum, Eric D. Marsh, Paul Howard
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-9 (2022)
Abstract Scientific advances in the understanding of the genetics and mechanisms of many rare diseases with previously unknown etiologies are inspiring optimism in the patient, clinical, and research communities and there is hope that disease-specifi
Externí odkaz:
https://doaj.org/article/912c5bda67e64e25b2ba4231d60e984b
Autor:
Nathan Denton, Monique Molloy, Samantha Charleston, Craig Lipset, Jonathan Hirsch, Andrew E. Mulberg, Paul Howard, Eric D. Marsh
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-4 (2021)
Abstract Data silos are proliferating while research and development activity explode following genetic and immunological advances for many clinically described disorders with previously unknown etiologies. The latter event has inspired optimism in t
Externí odkaz:
https://doaj.org/article/765210832e944959ba2a8c080ec7e704
Autor:
Melodi Harfouche, Priya S. Kishnani, Eva Krusinska, Jamie Gault, Sheela Sitaraman, Amanda Sowinski, Irina Katz, Stephanie Austin, Margi Goldstein, Andrew E. Mulberg
Publikováno v:
Journal of Patient-Reported Outcomes, Vol 4, Iss 1, Pp 1-11 (2020)
Abstract Background Patient-Reported Outcomes provide an opportunity for patients to establish dialogue with pharmaceutical or biotechnology companies about their health conditions without interpretation by a clinician or anyone else. However, Patien
Externí odkaz:
https://doaj.org/article/326ca216a52b4ce896d73b2b3dc9ca1a
Autor:
Nathan Denton, Andrew E. Mulberg, Monique Molloy, Samantha Charleston, David C. Fajgenbaum, Eric D. Marsh, Paul Howard
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-1 (2023)
Externí odkaz:
https://doaj.org/article/7624886844a3449395011d8c2f2dc551
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
Publikováno v:
Current Therapeutic Research, Vol 90, Iss , Pp 109-112 (2019)
ABSTRACT: The article, ''Questionable Industry-Sponsored Studies in Children and Adolescents in Slovenia'' provides an opportunity to discuss evolving US and EU legislative measures to improve the available clinical trial-derived pediatric data and p
Externí odkaz:
https://doaj.org/article/91615cd25622498ea33b6b19aec7a03c
Autor:
Timothy Cripps, Andrew E. Mulberg
Publikováno v:
Pediatric Inflammatory Bowel Disease ISBN: 9783031147432
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::441bf270d16b11c5d09a729d635a6672
https://doi.org/10.1007/978-3-031-14744-9_48
https://doi.org/10.1007/978-3-031-14744-9_48
Autor:
Andrew E. Mulberg, Laurie S. Conklin, Nicholas M. Croft, Anushree Parikh, Athos Bousvaros, Edwin DeZoeten, Marla Dubinsky, Francisco Javier Martin Carpi, Hankje Escher, Richard Vesely, Cecile Ollivier, Wallace Crandall, Collin Hovinga
Publikováno v:
Gastroenterology, 163(1), 77-83. W.B. Saunders
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4ad67411433358aaa46dea973f5a7715
https://pure.eur.nl/en/publications/4497e476-ee73-454d-b35c-857d1b061414
https://pure.eur.nl/en/publications/4497e476-ee73-454d-b35c-857d1b061414
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
Publikováno v:
Pancreas. 51(4)
We sought data on the validity, reliability, responsiveness, and feasibility of the coefficient of fat absorption (CFA) as a measure of pancreatic enzyme replacement therapy (PERT) efficacy in people with cystic fibrosis (pwCF) and reviewed the liter
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7490ff477a6fdbd715eb1dc4b9a91771
https://pubmed.ncbi.nlm.nih.gov/35695742
https://pubmed.ncbi.nlm.nih.gov/35695742