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Patients with undiagnosed and/or rare disorders frequently manifest dysmorphic and neurological features. There is a lack of information on the effectiveness of telehealth in the evaluation of these disorders. We thus compared an unassisted virtual physical examination (PE) with an in-person PE in undiagnosed individuals and also assessed participant telehealth satisfaction. Twenty-six individuals enrolled in the Undiagnosed Diseases Network study underwent an in-home synchronous virtual PE, and a subsequent in-person PE, by the same clinician. The participants completed surveys on telehealth usability and provider empathy. On PE, general appearance and craniofacial features showed near perfect agreement (κ = 0.81-1.00) between the telehealth and in-person evaluations. Specific components of the neurological examination demonstrated substantial agreement (speech, gait, coordination; κ > 0.61), whereas others had moderate agreement (muscle tone, strength; κ = 0.41-0.60), and a few had none to slight agreement (skin; κ < 0.20). Some systems could not be examined in the virtual PE. Importantly, features relevant to diagnosis or management were missed on the virtual PE in only 2/26 individuals. The participants were satisfied with the quality of the telehealth interaction, as well as empathy demonstrated by providers in the virtual interface. Telehealth is effective for PEs in undiagnosed diseases and is acceptable to affected individuals.
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http://dx.doi.org/10.1002/ajmg.a.63956 | DOI Listing |
Retin Cases Brief Rep
September 2025
Doheny Eye Institute, David Geffen School of Medicine, University of California, Los Angeles, California, USA.
Purpose: To report the examination and multimodal imaging findings of a patient with unilateral bull's eye maculopathy.
Methods: A retrospective chart review of a 77-year-old patient with unilateral bull's eye maculopathy who presented to a tertiary retinal practice was performed. The patient's history, visual acuity, examination and multimodal imaging findings over five years of follow-up were described.
Neurology
October 2025
Department of Neurology, Mayo Clinic, Rochester, MN.
Monoclonal gammopathy-associated myopathies (MGAMs) are rare yet treatable myopathies that occur in association with monoclonal gammopathies. These myopathies include light chain (AL) amyloidosis myopathy, sporadic late-onset nemaline myopathy (SLONM), scleromyxedema with associated myopathy, and newly reported monoclonal gammopathy-associated glycogen storage myopathy (MGGSM), including the vacuolar myopathy with monoclonal gammopathy and stiffness. All these 4 distinct subtypes of MGAMs typically present in patients aged 40 or older, frequently with a subacute onset of rapidly progressive proximal and axial muscle weakness.
View Article and Find Full Text PDFAm J Respir Cell Mol Biol
September 2025
Univ. of Pennsylvania, Medicine, Philadelphia, Pennsylvania, United States.
Lymphangioleiomyomatosis (LAM) is a rare lung disease caused by hyperactivation of the mechanistic/mammalian target of rapamycin 1 (mTORC1) growth pathway in a subset of mesenchymal lung cells. Histopathologically, LAM lesions have been described as immature smooth muscle-like cells positive for the immature melanocytic marker HMB45/PMEL/gp100 and phosphorylated ribosomal protein S6 (pS6). Advances in single cell sequencing (scRNA-seq) technology allowed us to group LAM cells according to their expression of cancer stem cell (CSC) genes and identify three clusters: a high CSC-like state (SLS), an intermediate state, and a low CSC-like inflammatory state (IS).
View Article and Find Full Text PDFMed Sci (Paris)
September 2025
Service des maladies de l'appareil digestif. Centre de compétence Maladies rares « Maladies inflammatoires des voies biliaires et hépatites autoimmunes », Hôpital Huriez, Lille, France.
Primary biliary cholangitis (PBC) is a rare disease for which management long consisted of a single treatment: ursodeoxycholic acid. In 2015-2016, this disease regained interest with the first studies on obeticholic acid (FXR agonist) and then on bezafibrate (PPAR agonist). Subsequently, over the past five years, significant progress has been made in the management of PBC.
View Article and Find Full Text PDFParasite
September 2025
Parasitology Department, São Paulo University, 1374 Av. Prof. Lineu Prestes, São Paulo, State of São Paulo 05508-000, Brazil.
Understanding why Diptera, such as mosquitoes and sand flies, feed on humans is crucial in defining them as vectors of diseases such as malaria, dengue fever, Zika virus, and leishmaniasis. Determining their attraction to humans (anthropophily) helps in assessing the risk of disease transmission, designing effective vector control strategies, and monitoring the effectiveness of existing control measures. An important question is whether they are specifically attracted to humans in preference to other mammals or whether there is something else at play.
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