Results 181 to 190 of about 48,580 (292)

Allogeneic hematopoietic cell transplantation is curative in CARMIL2 deficiency. [PDF]

J Hum Immun
Raedler J, Gothe F, Magg T, Peters P, Bronken Martinsen KH, Holzer U, Kiykim A, Kalyoncu Ucar A, Abraham RS, Rangarajan HG, NaserEddin A, Stepensky P, Zaidman I, Even-Or E, Kuehl JS, Graefe D, Bigley V, Tsilifis C, Fasth A, Mahlaoui N, Wegehaupt O, Speckmann C, Casanova JL, Klein C, Neven B, Béziat V, Lévy R, Hauck F.   +27 more
europepmc   +1 more source

‘We're not going to start lifting stones now…’: Stakeholder perspectives on the role of psychometric methods in outcome measurement

British Journal of Clinical Psychology, EarlyView.
Abstract Objectives Psychological outcome measures guide research and clinical decision‐making, yet many widely used tools were developed with limited psychometric rigour. Although advanced methods (e.g., item response theory, structural equation modelling) are now widely available, their added value in applied research remains uncertain and applied ...
David Byrne, Fiona Boland, Susan Brannick, Robert M. Carney, Pim Cuijpers, Alexandra L. Dima, Kenneth E. Freedland, Suzanne Guerin, David Hevey, Bishember Kathuria, Emma Wallace, Frank Doyle   +11 more
wiley   +1 more source

Maternal-Fetal Administration of Risdiplam Partially Rescues the SMNΔ7 Mouse Model of Spinal Muscular Atrophy. [PDF]

Ann Neurol
Sutton ER, Beauvais A, Yaworski R, McMillan HJ, Kothary R.   +4 more
europepmc   +1 more source

CRB1‐Associated Inherited Retinal Dystrophies: Prospective Natural History Study With 4 Years of Follow‐Up

Clinical &Experimental Ophthalmology, EarlyView.
ABSTRACT Background The lack of validated and sensitive clinical endpoints remains a major challenge in the design of gene therapy trials for inherited retinal dystrophies (IRDs). This prospective longitudinal cohort study describes the natural disease progression of IRDs caused by pathogenic mutations in the Crumbs homologue 1 (CRB1) gene, and ...
Jessica S. Karuntu, Xuan‐Thanh‐An Nguyen, Mays Talib, Mary J. van Schooneveld, Jan Wijnholds, Maria M. van Genderen, Jos Twisk, Nicoline E. Schalij‐Delfos, Caroline C. W. Klaver, Ralph J. Florijn, Jacoline B. ten Brink, Magda A. Smoor, L. Ingeborgh van den Born, Carel B. Hoyng, Alberta A. H. J. Thiadens, Camiel J. F. Boon   +15 more
wiley   +1 more source

Characterization and Validation of Long-Term Cultured TERT-Immortalized Human Wharton's Jelly-Derived Mesenchymal Stromal Cells. [PDF]

Stem Cell Rev Rep
Kim YE, Ahn SY, Lee YJ, Hwang S, Chang YS.   +4 more
europepmc   +1 more source

Characteristics of Emotional Support Provided by Adolescent Young Carers: A Qualitative Exploratory Study

Child &Family Social Work, EarlyView.
ABSTRACT Adolescent young carers (AYCs), aged 12–17, provide regular, unpaid support to a family member with a chronic illness or disability. Among the various forms of help they provide, emotional support (ES) plays a central role—demanding strong emotional and psychological investment yet remaining underexplored during adolescence, a period of ...
Céline Lacombe, Julie Glatz, Aurélie Untas   +2 more
wiley   +1 more source

Vestibular Evoked Myogenic Potentials (cVEMP and oVEMP) in Pregnancy: A Clinical Study. [PDF]

Audiol Res
Cakmak Karaer I, Sarıdogan E.
europepmc   +1 more source

Financially Coerced ‘Self‐Produced’ Child Sexual Exploitation Material: Narratives of Accountability

Child &Family Social Work, EarlyView.
ABSTRACT Perpetrators financially coerce children to ‘self‐produce’ child sexual exploitation material (CSEM). A harmful dominant narrative has historically blamed victims by framing this form of child sexual exploitation as ‘children engaging in sex work’ obfuscating the tactics of perpetrators.
Genevieve Bloxsom, Gemma McKibbin, Jennifer Davidson, Nick Halfpenny, Cathy Humphreys   +4 more
wiley   +1 more source

Radiographers' Experiences of Clinical Handover in Diagnostic Imaging: A Qualitative Study of Communication, Safety, and Organizational Influences. [PDF]

J Multidiscip Healthc
Makanjee CR, Kaur J, Hart R.
europepmc   +1 more source

Understanding Pathways to Recidivism Among Children in Türkiye: Insights From Social Workers on Needs, Neglect and Systemic Gaps

Child &Family Social Work, EarlyView.
ABSTRACT Türkiye has experienced a notable increase in juvenile delinquency in recent years, raising serious concerns about child welfare and juvenile justice systems. This qualitative study examines the socio‐economic and structural dimensions of recidivism based on the field experiences of 20 social workers working with children drawn into crime ...
Hüseyin Batman, Aykut Can Demirel, Murat Derin, Metin Gani Tapan   +3 more
wiley   +1 more source