Results 181 to 190 of about 5,265,871 (311)

Help-seeking patterns and funding strategies in patients with pulmonary arterial hypertension on phosphodiesterase-5 inhibitors: an orphan disease with effective but costly treatment. [PDF]

Singapore Med J, 2021
Lim Y, Maaroof SMB, Low TT, Kuntjoro I, Yip JW, Tay E.   +5 more
europepmc   +1 more source

Current state of medical care for patients with mycosis fungoides and Sézary syndrome at a German university hospital – a cross‐sectional study

JDDG: Journal der Deutschen Dermatologischen Gesellschaft, EarlyView.
Summary Background Studies on the state of medical care for patients with cutaneous T‐cell lymphoma (CTCL) are limited. To date, only secondary data analyses are available for Germany. Patients and Methods Cross‐sectional study conducted over a one‐year recruitment period (01 March 2024 to 28 February 2025) in the dermatological department of a German ...
Inga Hansen‐Abeck, Larissa Kölln, Finn Abeck, Glenn Geidel, Stefan W. Schneider, Nina Booken   +5 more
wiley   +1 more source

TB/HIV: An Orphan Disease? [PDF]

American Journal of Respiratory and Critical Care Medicine, 2011
James C M, Brust, Max R, O'Donnell, John Z, Metcalfe   +2 more
openaire   +2 more sources

The utility of the term ‘Rett‐like’ in relation to Rett syndrome: A systematic review

Developmental Medicine &Child Neurology, EarlyView.
Our findings show that ‘Rett‐like’ generally describes females with developmental disability, typically with no period of regression. We establish that there is a broad genetic landscape encompassing this disorder. Clinical utility of ‘Rett‐like’ is limited, with inadequate evidence of validity and diagnostic use of ‘Rett‐like’.
Anahita Khot, Daniel E Lumsden
wiley   +1 more source

Ethical dilemmas about orphan drugs for orphan diseases [PDF]

Journal of Paediatrics and Child Health, 2014
openaire   +4 more sources

Clonazepam repurposing in <i>ARID1B</i> patients through conventional RCT and N-of-1 trials: an experimental strategy for orphan disease development. [PDF]

J Med Genet
van der Sluijs PJ, Safai Pour K, Berends CL, Kruizinga MD, Müller AR, van Eeghen AM, Rodríguez-Girondo M, Juachon MJ, Steenbeek D, Cohen AF, Zuiker RGJA, Santen GWE.   +11 more
europepmc   +1 more source

[News Clip: Orphan Twins]

, 1960
Video footage from the WBAP-TV television station in Fort Worth, Texas, to accompany a news story about a couple who are waiting for the arrival of their new children, 2 orphan ...
WBAP-TV (Television station : Fort Worth, Tex.)
core  

Economic evaluation of the Akwenda Intervention Program for children and young people with cerebral palsy in rural Uganda: A trial‐based analysis of costs and outcomes

Developmental Medicine &Child Neurology, EarlyView.
Abstract Aim To relate the trial‐based costs of the Akwenda Intervention Program, a multi‐component, community‐based intervention for children and young people with cerebral palsy (CP) in rural Uganda, to multiple prespecified child and caregiver outcomes.
Elizabeth Asige, Eric Kintu, Gillian Saloojee, Godfrey Wanjala, Carin Andrews, Lukia H. Namaganda, Angelina Kakooza‐Mwesige, Diane L Damiano, Hans Forssberg   +8 more
wiley   +1 more source

Corrigendum: Abnormalities of thymic stroma may contribute to immune dysregulation in murine models of leaky severe combined immunodeficiency

Frontiers in Immunology
Francesca Rucci, Pietro Luigi Poliani, Stefano Caraffi, Tiziana Paganini, Elena Fontana, Silvia Giliani, Frederick W. Alt, Luigi Daniele Notarangelo   +7 more
doaj   +1 more source

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. [PDF]

Pharmaceutics
Szebeni J.
europepmc   +1 more source