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Acute effects of 50 mg additive oral sildenafil on invasive exercise haemodynamics in pulmonary vascular disease.
Tissue and systemic inflammation in dystrophic epidermolysis bullosa: a systematic review and meta-analysis.
COVID- 19 in patients affected by red blood cell disorders, results from the European registry ERN-EuroBloodNet.
Analysis of SMN protein in umbilical cord blood and postnatal peripheral blood of neonates with SMA: a rationale for prompt treatment initiation to prevent SMA development.
Efficacy of tranilast in preventing exacerbating cardiac function and death from heart failure in muscular dystrophy patients with advanced-stage heart failure: a single-arm, open-label, multicenter study.