Royal Manchester Children's Hospital

Molecular mechanisms underlying the effectiveness of triiodothyroacetic acid (Triac) in patients with resistance to thyroid hormone β (RTHβ): a multicentre, international, retrospective cohort study

Background Disease features in resistance to thyroid hormone beta (RTHβ), caused by THRB mutations, arise from insensitivity to thyroid hormone in tissues expressing thyroid hormone receptor (TR)β and from lifelong thyrotoxic effects in TRα1-expressing tissues, posing substantial...

Classification of Seasonal Allergic Rhinitis Patients by Prospective Patient‐Recorded Symptoms: The @ IT .2020 Project

BACKGROUND: Seasonal allergic rhinitis (SAR) has been so far classified according to frequency and severity by retrospective information based on the ARIA (Allergic Rhinitis and its Impact on Asthma) classification. Electronic symptom diaries (e-Diaries) are feasible tools to col...

Cross-cultural adaptation and validation of the Localised Scleroderma Quality of Life Instrument (LoSQI): an international study

Juvenile localised scleroderma (jLS) is a rare disease which impacts negatively on health-related quality of life (HRQoL). We aimed to undertake the cross-cultural adaptation and validation of a novel disease-specific HRQoL measure, the Localised Scleroderma Quality of Life Instr...