Results 171 to 180 of about 233,409 (358)

Report on the Muscular Dystrophy Campaign workshop: Exercise in neuromuscular diseases Newcastle, January 2002 [PDF]

, 2002
Michelle Eagle
openalex   +1 more source

FRAME: Framework for Real‐World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness – An Evaluation of Regulatory and Health Technology Assessment Decision Making

Clinical Pharmacology &Therapeutics, EarlyView.
Real‐World Evidence (RWE) is increasingly used in submissions to regulatory agencies and health technology assessment bodies (HTAbs) to support the efficacy and effectiveness of new medicines and indications. However, there is limited information on the RWE characteristics that impact its role in approval and reimbursement decisions.
Gianmario Candore, Claire Martin, Mack J. Mills, Annabel Suter, Anna Lloyd, Danitza Chavez‐Montoya, Diego Civitelli, Birgit Wolf, Paul Bolot, Juergen Wasem, Montse Soriano Gabarró, Panos G. Kanavos, Mark Sculpher   +12 more
wiley   +1 more source

The role of lung volume recruitment therapy in neuromuscular disease: a narrative review. [PDF]

Front Rehabil Sci, 2023
Sheers NL, O'Sullivan R, Howard ME, Berlowitz DJ.   +3 more
europepmc   +1 more source

Dysfunction in the Larynx and Its Surrounding Region Caused by Neuromuscular Diseases.

, 1991
Takemoto Shin, Tadatsugu Maeyama
openalex   +2 more sources

Motion of the Diaphragm in Patients with Chronic Obstructive Pulmonary Disease while Spontaneously Breathing versus during Positive Pressure Breathing after Anesthesia and Neuromuscular Blockade [PDF]

, 2002
Bruce Kleinman, Kerry Frey, Mark VanDrunen, Taqdees Sheikh, Donald DiPinto, Robert W. Mason, Theodore C. Smith   +6 more
openalex   +1 more source

Optimizing Patient Registries for Regulatory Decision Making ‐ Key Learnings From an HMA/EMA Multistakeholder Workshop

Clinical Pharmacology &Therapeutics, EarlyView.
The Joint Heads of Medicines Agencies and European Medicines Agency's (HMA/EMA) big data initiative paves the way for better integration of real‐world data, including data from patient registries, into regulatory decisions on medicines. This article focuses on the outcome of a two‐day multistakeholder workshop organized by EMA in 2024, which explored ...
Kelly Plueschke, Carla Jonker, Hanna Kankanen, Thorsten Vetter, Bruno Sepodes, Lutz Naehrlich, Jan Hillert, Gracy Crane, Sabine Straus, Paolo Foggi, Simona Martin, Christina Kyriakopoulou, Peter Mol, Francesca Day, Kieran Breen, Neil Bennett, Mencia de Lemus Belmonte, Simon Bennett, Patrice Verpillat, Kit C. B. Roes, Ana Cochino, Franz Schaefer, Jesús María Hernández‐Rivas, Patricia McGettigan, Peter Arlett   +24 more
wiley   +1 more source

A scalable human iPSC-based neuromuscular disease model on suspended biobased elastomer nanofiber scaffolds. [PDF]

Biofabrication, 2023
Cheesbrough A, Harley P, Riccio F, Wu L, Song W, Lieberam I.   +5 more
europepmc   +1 more source

Human T cell leukaemia virus type I associated neuromuscular disease causing respiratory failure [PDF]

, 2002
Edward Littleton
openalex   +1 more source

Role of selenium in the pathophysiology of cardiorenal anaemia syndrome

ESC Heart Failure, Volume 12, Issue 2, Page 770-780, April 2025.
Abstract Chronic kidney disease (CKD) and cardiovascular disease (CVD) have multiple bidirectional mechanisms, and anaemia is one of the critical factors that are associated with the progression of the two disorders [referred to as cardiorenal anaemia syndrome (CRAS)].
Shigeyuki Arai, Minoru Yasukawa, Shigeru Shibata   +2 more
wiley   +1 more source

P1112: TREATING BING-NEEL SYNDROME USING A TAILORED APPROACH: EXPERIENCE OF A SPECIALIST NEUROHAEMATOLOGY CLINIC

HemaSphere, 2022
J. Khwaja, D. Smyth, A. Rismani, C. Hoskote, C. Kyriakou, M. P. Lunn, S. D’Sa   +6 more
doaj   +1 more source