Results 201 to 210 of about 5,265,871 (311)

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF‐McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Mark W. Skinner, Manuela Albisetti, Jesús Ardila, Jan Astermark, Jan Blatny, Manuel Carcao, Pratima Chowdary, Nathan T. Connell, Miguel Crato, Yesim Dargaud, Roseline d'Oiron, Amy L. Dunn, Miguel A. Escobar, Carmen Escuriola‐Ettingshausen, Emna Gouider, Annie Harroche, Cedric Hermans, Victor Jimenez‐Yuste, Radoslaw Kaczmarek, Gili Kenet, Liane Khoo, Robert Klamroth, Florian Langer, David Lillicrap, Johnny Mahlangu, Christoph Male, Tadashi Matsushita, Sandrine Meunier, Wolfgang Miesbach, Beatrice Nolan, Johannes Oldenburg, Brian O'Mahony, Margareth Ozelo, Glenn F. Pierce, Gloria Ramos, Michael Recht, Olivia Romero‐Lux, Dawn Rotellini, Rita C. Santoro, Tammuella C. Singleton, Alok Srivastava, Sophie Susen, Kate Talks, Huyen Tran, Leonard A. Valentino, Jerzy Windyga, Renchi Yang, Maria Elisa Mancuso, also on behalf of AICE, AHAD‐AP, AHCDC, ATHN, CLAHT, EHC, French CRH & MHEMO, HTCCNC, NBDF, PEDNET, UKHCDO, WFH   +59 more
wiley   +1 more source

New orphan disease therapies from the proteome of industrial plasma processing waste- a treatment for aceruloplasminemia. [PDF]

Commun Biol
Zanardi A, Nardini I, Raia S, Conti A, Ferrini B, D'Adamo P, Gilberti E, DePalma G, Belloli S, Belloli S, Monterisi C, Coliva A, Rainone P, Moresco RM, Mori F, Zurlo G, Scali C, Natali L, Pancanti A, Giovacchini P, Magherini G, Tovani G, Salvini L, Cicaloni V, Tinti C, Tinti L, Lana D, Magni G, Giovannini MG, Gringeri A, Caricasole A, Alessio M.   +31 more
europepmc   +1 more source

Correction: Szebeni, J. Expanded Spectrum and Increased Incidence of Adverse Events Linked to COVID-19 Genetic Vaccines: New Concepts on Prophylactic Immuno-Gene Therapy, Iatrogenic Orphan Disease, and Platform-Inherent Challenges. <i>Pharmaceutics</i> 2025, <i>17</i>, 450. [PDF]

Pharmaceutics
Szebeni J.
europepmc   +1 more source

Real‐World Outcomes of Prophylaxis With rIX‐FP in Germany: A Prospective, Non‐Interventional Study in Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Efficacy and safety of albutrepenonacog alfa (rIX‐FP) in people with haemophilia B (PwHB) has been previously demonstrated in clinical trials. However, real‐world data insights are required. Aim To describe real‐world effectiveness and tolerability of rIX‐FP in PwHB in Germany.
Johannes Oldenburg, Swee Wenning, Susanne Holzhauer, Wolfgang Miesbach, Robert Klamroth, Thomas Linhoff, Patrick Sommerer, Christian Pfrepper, Martin Olivieri, all study investigators   +9 more
wiley   +1 more source

Orphan disease: Cherubism, optic atrophy, and short stature. [PDF]

Indian J Radiol Imaging, 2018
Jeevanandham B, Ramachandran R, Dhanapal V, Subramanian I, Sai V.   +4 more
europepmc   +1 more source

Patient Perspectives in Haemophilia A: Unmet Needs of Individuals Receiving Factor and Non‐Factor Treatments

Haemophilia, EarlyView.
ABSTRACT Introduction There is limited qualitative published real‐world data on treatment patterns among people with severe haemophilia A (PwSHA), particularly those with unmet needs. Aim This qualitative study aimed to explore the impact of taking prophylactic extended half‐life factor VIII (EHL‐FVIII) or emicizumab on psychosocial and physical ...
Nana Kragh, Veruska Carboni, Duygu Bozkaya, Idaira Rodriguez‐Santana, Claudia Mighiu, Bethany Franks, Arnoud Plat, Randall Curtis   +7 more
wiley   +1 more source

Anaesthesia and orphan disease: airway and anaesthetic management in Huntington's disease. [PDF]

BMJ Case Rep, 2017
Nguyen PT, Meeks D, Liotiri D.
europepmc   +1 more source

Orcein is a more sensitive stain than EVG for detecting large vessel (venous) invasion in colorectal cancer

Histopathology, EarlyView.
Orcein is more sensitive than EVG for detecting intramural and extramural large vessel (venous) invasion than EVG and H&E in colorectal cancer. Background Large vessel invasion (VI) is a well‐established adverse prognostic factor in colorectal cancer (CRC).
Timothy Law, Won‐Tak Choi, Sarah E. Umetsu, Kwun Wah Wen   +3 more
wiley   +1 more source

Novel CHM mutations in Polish patients with choroideremia - an orphan disease with close perspective of treatment. [PDF]

Orphanet J Rare Dis, 2018
Skorczyk-Werner A, Wawrocka A, Kochalska N, Krawczynski MR.   +3 more
europepmc   +1 more source

Issues Surrounding Orphan Disease and Orphan Drug Policies in Europe

An orphan disease is a disease with a very low prevalence. Although there are 5000-7000 orphan diseases, only 50 orphan drugs (i.e. drugs developed to treat orphan diseases) were marketed in the EU by the end of 2008. In 2000, the EU implemented policies
Lut Mergaert, Christel Fostier, Steven Simoens, Alain Denis, Irina Cleemput   +4 more
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