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BridgeBio Pharma : BBIO – Acoramidis AG10 TTR – ISA 2024 – Acoramidis treatment related increase in serum TTR is associated with lower cardiovascular mortality in ATTR CM

Treatment-Related Early Increase in Serum TTR is Associated With Lower Cardiovascular Mortality in ATTR-CM: Insights From ATTRibute-CM Amrut.

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BridgeBio Pharma : BBIO – Acoramidis AG10 TTR – ISA 2024 – Higher risk of mortality in previously hospitalized patients

Higher Risk of Mortality in Previously Hospitalized Patients: Insights From ATTRibute-CM Ahmad Masri1, Michele Emdin2, Keyur Shah3, Kunal Bhatt4,.

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Neurocrine Biosciences (NBIX) Presented Baseline Data from the CAHtalyst Program in CAH and Study Data for Modified-Release Hydrocortisone in Primary Adrenal Insufficiency and CAH at ECE 2024

Neurocrine Biosciences (NBIX) Presented Baseline Data from the CAHtalyst Program in CAH and Study Data for Modified-Release Hydrocortisone in Primary Adrenal Insufficiency and CAH at ECE 2024
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Study Design and Baseline Characteristics of Prespecified, Exploratory Pooled Analysis to Investigate Effect of Finerenone on Cardio-Kidney Outcomes in Patients with CKD and Type 2 Diabetes and/or Heart Failure Shared at Heart Failure 2024 Congress

Study Design and Baseline Characteristics of Prespecified, Exploratory Pooled Analysis to Investigate Effect of Finerenone on Cardio-Kidney Outcomes in Patients with CKD and Type 2 Diabetes and/or Heart Failure Shared at Heart Failure 2024 Congress
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MeiraGTx : Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction – The AQUAx Study

Results of a Phase 1, Open-label,Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction -.

United statesAmericanMichaelt brennanDose escalation studyGene therapyLate xerostomiaParotid gland hypofunctionInduced xerostomiaUnmet medical needStudy designSymptom inventoryChange questionnaireBaseline characteristicsTotal xerostomia questionnaireDry mouthRelatedtreatment emergent adverse events