American journal of respiratory and critical care medicine
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Am. J. Respir. Crit. Care Med. · Nov 2010
Randomized Controlled TrialAtaluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Nonsense (premature stop codon) mutations in mRNA for the cystic fibrosis transmembrane conductance regulator (CFTR) cause cystic fibrosis (CF) in approximately 10% of patients. Ataluren (PTC124) is an oral drug that permits ribosomes to readthrough premature stop codons in mRNA to produce functional protein. ⋯ In children with nonsense mutation CF, ataluren can induce functional CFTR production and is well tolerated.
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Am. J. Respir. Crit. Care Med. · Nov 2010
Randomized Controlled TrialImatinib in pulmonary arterial hypertension patients with inadequate response to established therapy.
Pulmonary arterial hypertension (PAH) is a progressive condition with a poor prognosis. Platelet-derived growth factor receptor (PDGFR) signaling plays an important role in its pathobiology. ⋯ These data from a Phase II study are consistent with imatinib being well tolerated in patients with PAH, and provide proof of concept for further studies evaluating its safety, tolerability, and efficacy in PAH. Clinical trial registered with www.clinicaltrials.gov (NCT00477269).
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Am. J. Respir. Crit. Care Med. · Nov 2010
Randomized Controlled TrialPulmonary vascular dysfunction is associated with poor outcomes in patients with acute lung injury.
Despite the recognition that acute lung injury (ALI) can elevate pulmonary artery (PA) pressure and right ventricular afterload, the impact of pulmonary vascular dysfunction on outcomes of these patients is not well defined. ⋯ Pulmonary vascular dysfunction is common in ALI, and is independently associated with poor outcomes. Future trials targeting pulmonary vascular dysfunction may be indicated.