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Results of a non-randomized, open-label phase I study evaluating the novel immunomodulatory peptide TCP-25 for treatment of dystrophic epidermolysis bullosa.
Fatigue in patients with syndromic heritable thoracic aortic disease: a systematic review of the literature and a qualitative study of patients' experiences and perceptions.
🥇 Revisão sistemáticaFat infiltration in the thigh muscles is associated with symptomatic spinal stenosis and reduced physical functioning in adults with achondroplasia.
Patient involvement in rare diseases research: a scoping review of the literature and mixed method evaluation of Norwegian researchers' experiences and perceptions.
📖 RevisãoExperienced fatigue in people with rare disorders: a scoping review on characteristics of existing research.
📖 Revisão