Exercise capacity (peak VO 2 ) assessed by CPET : meaningful improvement in 2 of 3 patients (worsened on ERT natural history study).Cardiac contractility (GLS) assessed on ECHO : meaningful improvement in 3 of 3 patients (GLS worsened on ERT natural history study).Cardiac biopsy available from one patient in the INGLAXA-2 trial.Quality of life status – Change from baseline at 12 months in Kansas City Cardiomyopathy Questionnaire (KCCQ).Exercise capacity – Change from baseline at 12 months in cardiopulmonary exercise testing (peak VO 2 by CPET). ![]() Left ventricular contractility – Change from baseline at 12 months in global longitudinal strain on echocardiography (GLS on ECHO).Assessed FDA-recommended pivotal trial cardiac endpoints in three patients who completed 12 months of follow-up (cut-off date of December 5, 2022) including:.Enrolled six patients, each treated with 1E13 vg/kg of 4D-310 and a prophylactic corticosteroid immunosuppressive regimen.Treated patients in three geographies: INGLAXA-1 (U.S.) and INGLAXA-2 (Taiwan and Australia). ![]()
0 Comments
Leave a Reply. |
Details
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |