Results 191 to 200 of about 71,497 (297)

Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine‐learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the ...
Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel   +9 more
wiley   +1 more source

Elevated Urinary Titin in Adult Spinal Muscular Atrophy: A Multicenter, Cross-Sectional Observational Study. [PDF]

Neurol Int
Sipos A, Ripszám ER, Molnár JM, Grosz Z, Boczán J, Altorjay MB, Dézsi L, Csáti A, Babarczy K, Kovács N, Hajdú N, Pál E.   +11 more
europepmc   +1 more source

Review of published research on primary dysautonomia of domestic animals

Veterinary Record, EarlyView.
Abstract An article published in 1992 by Marion M. Pollin and I.R. Griffiths reviewed the topic of primary dysautonomias of the autonomic nervous system of unidentified origin in multiple domestic species, specifically cats, dogs and horses. Thirty‐one years later, we appear no closer to identifying the causal agents of these strikingly similar ...
Tanith Harte, David Smith, Jo Moore, Beth Wells   +3 more
wiley   +1 more source

Classifying neurogenic dysphagia as a movement disorder. [PDF]

Brain
Jones CA, Huckabee ML, Malandraki GA, Paydarfar D.   +3 more
europepmc   +1 more source

Chinese Clinical Practice Guidelines for Auditory Neuropathy (gCAN)

World Journal of Otorhinolaryngology - Head and Neck Surgery, EarlyView.
ABSTRACT Auditory neuropathy (AN) is an auditory disorder that affects the function of the auditory pathway. An increasing number of AN cases have been identified with the revelation of the underlying mechanisms, the advancements of diagnostic and detecting techniques.
Chinese Multi‐Center Research Collaborative Group on Clinical Diagnosis and Intervention of Auditory Neuropathy; Editorial Board of Chinese Journal of Otorhinolaryngology Head and Neck Surgery; Society of Otorhinolaryngology Head and Neck Surgery, Chinese Medical Association; Society of Audiology and Vestibular Medicine, China International Exchange and Promotive Association for Medical and Health Care, Hong‐Yang Wang, Guo‐Hui Nie, Xiao‐Nan Wu, Ding‐Jun Zha, Tao Shi, Hai‐Bo Shi, Yong Feng, Wen‐Yan Li, Hai‐Hong Liu, Qing Zhang, Xiao‐Wei Chen, Xia Gao, Yong‐Xin Li, Lu Ma, Jin Li, Dan‐Yang Li, Xiao‐Long Zhang, Meng‐Tao Song, Xin Zhou, Lan Lan, Lan Yu, Meng‐Qian Zhang, Wei Shi, Fen Xiong, Lin‐Yi Xie, Guo‐Hui Chen, Kai‐Li Wu, Xiao‐Qian Ren, Qiu‐Jing Zhang, Ming‐Hui Zhao, Zi‐Yi Chen, Fei Ji, Jiao Zhang, Jing Guan, Da‐Yong Wang, Ren‐Jie Chai, Xin Jin, Hua‐Wei Li, Shi‐Ming Yang, Shan‐Kai Yin, Qiu‐Ju Wang   +43 more
wiley   +1 more source

Single Nucleotide <i>SMN1</i> Variants in a Cohort of Individuals With Spinal Muscular Atrophy. [PDF]

Neurol Genet
Rimoldi M, Magri F, Meneri M, Gagliardi D, Ada Sansone V, Albamonte E, Ottoboni L, Comi GP, Mercuri E, Tiziano FD, Ronchi D, Corti S.   +11 more
europepmc   +1 more source

Treatment of Neurogenic Voice Disorders

World Journal of Otorhinolaryngology - Head and Neck Surgery, EarlyView.
ABSTRACT This overview serves as a foundational resource for clinicians caring for neurologically complex patients presenting with voice complaints. Neurogenic voice disorders are diverse in their clinical presentations and therapeutic approaches. A thorough medical history, including family history, detailed laryngeal examination, voice assessments ...
Mausumi Syamal
wiley   +1 more source

Comorbidities in spinal muscular atrophy and their impact on the course of the underlying disease: a real-life observational study. [PDF]

BMC Musculoskelet Disord
Błauciak M, Ubysz J, Wieczorek M, Koszewicz M.   +3 more
europepmc   +1 more source

‘Expression is power’: Gender, residual culture and political aspiration at the Cumnock School of Oratory, 1870–1900

Gender &History, EarlyView.
Abstract This article investigates the ways in which late‐nineteenth‐century students at Northwestern University's Cumnock School of Oratory mobilised elocution training and parlour performance to foster mixed‐gender public discourse. I use student publications to reconstruct parlour meetings in which women and men adapted traditions of conversational ...
Fiona Maxwell
wiley   +1 more source

Imaging of Abdominal Complications in Children With Acute Lymphoblastic Leukaemia

Journal of Medical Imaging and Radiation Oncology, EarlyView.
ABSTRACT Acute lymphoblastic leukaemia (ALL) is the most common paediatric malignancy and remains one of the most common causes of cancer‐related death in children and adolescents. Five‐year overall survival rates now exceed 90% with current multidrug chemotherapeutic regimens. This improvement, coupled with the toxicity of chemotherapy, has led to the
Luke R. Holmes, Rishi S. Kotecha, Derek J. Roebuck   +2 more
wiley   +1 more source