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Navigating adulthood with PKU: metabolic outcomes, quality of life, and mental health 4.5 years post-transition.
Patient and caregiver experiences with a patient-support program for setmelanotide treatment of patients with Bardet-Biedl syndrome.
Acute effect of acetazolamide on exercise haemodynamics in patients with pulmonary arterial and chronic thromboembolic pulmonary hypertension: a randomised controlled trial.
Clinical characteristics and prognosis of amyopathic dermatomyositis patients with interstitial lung disease: insights from a retrospective cohort.
Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments.