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Background: The rarity that is inherent in rare disease (RD) often means that patients and parents of children with RDs feel uniquely isolated and therefore are unprepared or unsupported in their care. To overcome this isolation, many within the RD community turn to the internet, and social media groups in particular, to gather useful information about their RDs. While previous research has shown that social media support groups are helpful for those affected by RDs, it is unclear what these groups are particularly useful or helpful for patients and parents of children with RDs.

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Synthesis and Evaluation of a Bifunctional Chelator for Thorium-227 Targeted Radiotherapy.

J Med Chem

January 2025

Chemical Sciences Division, Lawrence Berkeley National Laboratory, Berkeley, California 94720, United States.

Thorium-227 (Th) is an α-emitting radionuclide currently under investigation for targeted alpha therapy. Available chelators used for this isotope suffer from challenging multistep syntheses. Here, we present the synthesis and preclinical evaluation of a novel bifunctional chelator, SCN-Bn-DOTHOPO, which contains an isothiocyanate group that is suitable for conjugation to biological molecules.

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This interview article explores how British anthropologist Tim Ingold's work can inspire innovation in mental health and the psy disciplines. Ingold critiques dominant biomedical and individualistic approaches, arguing for the importance of caring attentiveness and abolishing dichotomies like those between surface and depth, when engaging with people to understand and assist them. Instead, he suggests viewing human existence as correspondences with environmental, social, and relational others.

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Basic Science and Pathogenesis.

Alzheimers Dement

December 2024

Barrow Neurological Institute, Phoenix, AZ, USA; Arizona State University, Tempe, AZ, USA.

Background: TDP-43 is an RNA binding protein that is a pathological hallmark of multiple neurodegenerative diseases including Amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and Alzheimer's disease (AD). The frequency of observed TDP-43 pathology is estimated at 97% in ALS, 45% in FTD and 40-57% in AD and is characterized by a mislocalization of TDP-43 from the nucleus to the cytoplasm. Indeed, TDP-43 is the third most common proteinopathy in AD, behind only Amyloid beta and Tau.

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Basic Science and Pathogenesis.

Alzheimers Dement

December 2024

Indiana University, Indianapolis, IN, USA.

Background: Preclinical testing in animal models is a critical component of the drug discovery process. Over the past three decades hundreds of interventions have demonstrated preclinical efficacy for ameliorating cognitive impairments in animal models; however, none have translated to efficacy in Alzheimer's disease (AD) clinical trials. This lack of translation suggests that there are issues with the animal models employed, the preclinical assays, and poor scientific rigor and reproducibility during execution.

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