Results 71 to 80 of about 65,249 (309)

The 9th International RASopathies Symposium

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT The RASopathies are a group of congenital disorders with overlapping clinical manifestations that are caused by pathogenic germline or early somatic variants that result in the hyperactivation of the RAS/mitogen‐activated protein kinase (MAPK) signaling pathway.
Pau Castel, Lisa Schoyer, Beth Stronach, Raya Bogdanova, Anton M. Bennett, Jaishri Blakeley, Miriam Bornhorst, Tammy Bowers, Saskia M. Brachmann, Emma Burkitt‐Wright, Kathryn Chatfield, Alessandro De Luca, Khalil El‐Chammas, Abdul Elkadri, John E. Fortunato, Bruce D. Gelb, Anne Goriely, Karen Gripp, Kassidy Grover, Lindsay Homan, Kenneth A. Kern, Maija Kiuru, Charles (Chuck) Lawson, Yong‐Seok Lee, Frank McCormick, Gina Ney, Cristina Nuevo‐Tapioles, Sara Pardej, Elizabeth I. Pierpont, Julia Plank, Nancy Ratner, Katherine A. Rauen, J. Elliott Robinson, Les Rogers, Sarah E. Sheppard, Keir Shiels, David Stevenson, Dagmar Tiemens, Matthew Traylor, K. Nicole Weaver, Marielle Yohe, Tamar Green   +41 more
wiley   +1 more source

Rheumotologitsts’ view on the use of hydroxychloroquine to treat COVID-19

Emerging Microbes and Infections, 2020
The current pandemic coronavirus disease 2019 (COVID-19) caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) calls urgently for effective therapies.
Xiaoxuan Sun, Yicheng Ni, Miaojia Zhang
doaj   +1 more source

Outlook Magazine, Autumn 2013 [PDF]

, 2013
https://digitalcommons.wustl.edu/outlook/1190/thumbnail ...

core   +1 more source

Evidence and Extrapolation: Mechanisms for Regulating Off-Label Uses of Drugs and Devices [PDF]

, 2014
A recurring, foundational issue for evidence-based regulation is deciding whether to extend governmental approval from an existing use with sufficient current evidence of safety and efficacy to a novel use for which such evidence is currently lacking ...
Abbott, Ryan, Ayres, Ian
core   +3 more sources

Efficacy and safety of rasburicase, a recombinant urate oxidase (Elitek™), in the management of malignancy-associated hyperuricemia in pediatric and adult patients: final results of a multicenter compassionate use trial [PDF]

Leukemia, 2004
The recombinant urate oxidase, rasburicase (Elitek, Sanofi-Synthelabo, Inc.), has recently received regulatory approval for the prevention and treatment of hyperuricemia in children with leukemia, lymphoma, and solid tumors. Prior to approval, 682 children and 387 adults in the US and Canada received rasburicase on compassionate-use basis.
S, Jeha, H, Kantarjian, D, Irwin, V, Shen, S, Shenoy, S, Blaney, B, Camitta, C-H, Pui   +7 more
openaire   +2 more sources

Resolution of Refractory Multifocal Atrial Tachycardia in Costello Syndrome Using Trametinib: A Case Supporting MEK Inhibitors as Targeted, Specific Antiarrhythmic

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Arrhythmias affect approximately half of patients with Costello syndrome (CS, OMIM # 218040), with non‐reentrant atrial tachycardia being the most common. This case describes an infant with Costello syndrome carrying the pathogenic HRAS c.34G>A (p.G12S) variant who developed early‐onset, drug‐refractory multifocal atrial tachycardia (MAT ...
Vanina Taliercio, Annabelle Wilcox, Stacey Cole, Josue Flores Daboub, José E. Morales Moreno, Kasey G. Andrews, S. Yukiko Asaki, Thomas A. Pilcher, Martin Tristani‐Firouzi, Mary C. Niu, David Viskochil, Benjamin Hammond   +11 more
wiley   +1 more source

Triple peptide vaccination as consolidation treatment in women affected by ovarian and breast cancer: clinical and immunological data of a phase I/II clinical trial [PDF]

, 2016
Vaccination with priming and expansion of tumour reacting T cells is an important therapeutic option to be used in combination with novel checkpoint inhibitors to increase the specificity of the T cell infiltrate and the efficacy of the treatment. In
Antonilli M, BARCHIESI, GIACOMO, BELLATI, FILIPPO, BENEDETTI PANICI, PIERLUIGI, CAPONNETTO, SALVATORE, Iadarola R, NAPOLETANO, Chiara, NUTI, Marianna, PIERELLI, LUCA, RAHIMI KOSHKAKI, HASSAN, RUGHETTI, Aurelia, RUSCITO, ILARY, VISCONTI, Valeria, ZIZZARI, ILARIA GRAZIA   +13 more
core   +1 more source

Cost comparison of patients with 3-level artificial total lumbar disc replacements versus 360° fusion at 3 contiguous lumbar vertebral levels: an analysis of compassionate use at 1 site of the US investigational device exemption clinical trial [PDF]

SAS Journal, 2010
We sought to evaluate the difference between hospital service costs of 2 treatment options for patients diagnosed with 3-level degenerative disc disease (DDD) in the lumbar spine. In this retrospective analysis, itemized billing records of hospital stay for patients with 3-level DDD treated with artificial disc replacement (ADR) were compared with ...
Buttacavoli, Frank A., Delamarter, Rick B., Kanim, Linda E.A.   +2 more
openaire   +2 more sources

Superannuation Reimagined: Moving Beyond the Origins to an Indigenous Focus

Australian Journal of Social Issues, EarlyView.
ABSTRACT Retirement income systems, such as superannuation, are meant to be non‐discriminatory and consider disadvantage faced by members of society. There are significant differences between the life expectancies of Indigenous and non‐Indigenous peoples. The gap in life expectancies is not considered when determining when Indigenous peoples can retire.
Levon Ellen Blue, Peter Anderson, Mark Brimble   +2 more
wiley   +1 more source

First clinical experience of isatuximab safety and tolerability in relapsed and refractory multiple myeloma: real-world data from a compassionate use program in Germany

Frontiers in Hematology
Therapy for relapsed and refractory multiple myeloma (RRMM) remains challenging. While monoclonal antibodies against CD38 combined with pomalidomide have demonstrated efficacy in clinical trials, real-world data remain sparse.
Theo Leitner, Cyrus Khandanpour, Cyrus Khandanpour, Knut Wendelin, Fuat Oduncu, Christoph Kimmich, Ralph Naumann, Miriam Kull, Hartmut Goldschmidt, Martin Ehmer, Claudia Kiewitz, Hans Salwender   +11 more
doaj   +1 more source