The BLOOM clinical study is enrolling males with Fragile X Syndrome ages 13 to 45 to see if the investigational medicine, MRM-3379, can safely and effectively help improve certain symptoms of Fragile X Syndrome.
The VANTAGE clinical study is seeking patients ages 18 and older with PBC to learn if the investigational medication, volixibat, can safely and effectively reduce itching caused by PBC.
A clinical study, also called a clinical trial, is a research test to see if an investigational medicine is safe and effective in human volunteers. It compares the investigational medicine to a placebo (a treatment with no active ingredients). These medicines have not been fully approved by the FDA (Food and Drug Administration) or other health organizations. However, they can still be given to people as part of the study. Participants play a vital role in advancing medicine for current and future generations.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company conducting clinical trials to investigate potential treatments for rare diseases. We’re committed to putting the needs of patients and families at the center of our research by working alongside healthcare professionals to develop investigational therapies that may help rare disease communities.
For more information about Mirum, please visit MirumPharma.com.
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We offer the convenient option of allowing you to participate in our studies from the comfort of your own home. A healthcare professional will monitor you through a mix of telemedicine and home visits. The study doctor’s role is limited to the clinical study. While in the study, you will continue your routine medical care and see your regular doctors for check-ups and prescriptions.