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Mirum Pharmaceuticals (MIRM) Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum s LIVMARLI

Mirum Pharmaceuticals (MIRM) Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum s LIVMARLI
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Binitam kamathBettina hansenMirum pharmaceuticals incAlagille syndrome allianceGlobal alagille allianceStudy groupMirum pharmaceuticalsStaff physicianSenior associate scientistSick childrenPam vigClinical biostatistics

Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum s LIVMARLI -February 29, 2024 at 08:35 am EST

- Results demonstrate 70% risk reduction for long-term clinical outcomes in patients with Alagille syndrome treated with LIVMARLI - Data are the first to demonstrate a six-year transplant-free.

Binitam kamathBettina hansenAlagille syndrome allianceGlobal alagille allianceMirum pharmaceuticals incStudy groupEuropean commissionDrug administrationMirum pharmaceuticalsStaff physicianSenior associate scientistSick childrenPam vigClinical biostatisticsBreakthrough therapyFat soluble vitamin

Breaking Barriers in Childhood Rare Disease Trial Conduct

Breaking Barriers in Childhood Rare Disease Trial Conduct
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Shannon vandrielBinita kamathGlobal alagille allianceAlagille syndrome allianceIpsen pharmaceuticalsDivision of gastroenterologyMirum pharmaceuticals to sickkids foundationSick childrenSenior associate scientistStem cell biologyPrincipal investigatorStaff physicianMirum pharmaceuticals

Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum s LIVMARLI

Mirum Pharmaceuticals, Inc. today announced that Hepatology published an analysis demonstrating a statistically significant improvement in six-year event-free and transplant-free survival in patients with Alagille syndrome (ALGS) treated with.

Bettina hansenBinitam kamathStudy groupExchange commissionDrug administrationAlagille syndrome allianceMirum pharmaceuticals incGlobal alagille allianceEuropean commissionProduct serviceAlagille syndrome treatedStaff physicianSenior associate scientistSick childrenPam vigClinical biostatistics

SickKids study offers new path for pediatric rare disease clinical trials

Using a novel methodology, researchers at The Hospital for Sick Children (SickKids) are the first in pediatric research to use data from an international real-world cohort to overcome the barriers associated with conducting randomized clinical trials in children with rare diseases.

Shannon vandrielBinita kamathIpsen pharmaceuticalsGlobal alagille allianceAlagille syndrome allianceMirum pharmaceuticals to sickkids foundationDivision of gastroenterologySick childrenSenior associate scientistStem cell biologyPrincipal investigatorStaff physicianMirum pharmaceuticals