2023
Effects of sotatercept on lung diffusion capacity and blood gases in patients with pulmonary arterial hypertension
Olsson K, Fuge J, Park D, Kamp J, Brod T, Harrigfeld B, Schupp J, Hoeper M. Effects of sotatercept on lung diffusion capacity and blood gases in patients with pulmonary arterial hypertension. European Respiratory Journal 2023, 62: 2301070. PMID: 37474157, DOI: 10.1183/13993003.01070-2023.Peer-Reviewed Original ResearchConceptsPulmonary arterial hypertensionLung diffusion capacityArterial hypertensionDiffusion capacityRight heart functionRight ventricular afterloadSmall pulmonary vesselsVentricular afterloadBlood gasesPulmonary vesselsHeart functionRare diseaseHypertensionPatientsSotaterceptProgressive increaseSymptomsVariable alterationsDisease symptomsGas exchangeAfterloadDiseaseSeverity
2020
Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy
Jones SA, McGovern M, Lidove O, Giugliani R, Mistry PK, Dionisi-Vici C, Munoz-Rojas MV, Nalysnyk L, Schecter AD, Wasserstein M. Clinical relevance of endpoints in clinical trials for acid sphingomyelinase deficiency enzyme replacement therapy. Molecular Genetics And Metabolism 2020, 131: 116-123. PMID: 32616389, DOI: 10.1016/j.ymgme.2020.06.008.Peer-Reviewed Original ResearchConceptsAcid sphingomyelinase deficiencyDisease burdenLipid profilePrevalent clinical featuresRespiratory-related complicationsAtherogenic lipid profileAbnormal lipid profileProgressive lung diseaseLung diffusion capacityEnzyme replacement therapyRare lysosomal storage disorderDiverse disease spectrumDegree of abnormalityNiemann-Pick diseaseLysosomal storage disorderLung functionClinical featuresClinical parametersReplacement therapyChronic formClinical burdenLung diseaseNeurovisceral diseaseSpleen volumeLiver fibrosis
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