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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 Kamath , Bettina Hansen , Mirum Pharmaceuticals Inc , Alagille Syndrome Alliance , Global Alagille Alliance , Study Group , Mirum Pharmaceuticals , Staff Physician , Senior Associate Scientist , Sick Children , Pam Vig , Clinical Biostatistics ,

Breaking Barriers in Childhood Rare Disease Trial Conduct

Breaking Barriers in Childhood Rare Disease Trial Conduct
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Shannon Vandriel , Binita Kamath , Global Alagille Alliance , Alagille Syndrome Alliance , Ipsen Pharmaceuticals , Division Of Gastroenterology , Mirum Pharmaceuticals To Sickkids Foundation , Sick Children , Senior Associate Scientist , Stem Cell Biology , Principal Investigator , Staff Physician , Mirum Pharmaceuticals ,

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 Vandriel , Binita Kamath , Ipsen Pharmaceuticals , Global Alagille Alliance , Alagille Syndrome Alliance , Mirum Pharmaceuticals To Sickkids Foundation , Division Of Gastroenterology , Sick Children , Senior Associate Scientist , Stem Cell Biology , Principal Investigator , Staff Physician , Mirum Pharmaceuticals ,