At Mirum Pharmaceuticals, our work begins with a simple belief: people living with rare conditions deserve urgency, commitment, and meaningful scientific progress.
Mirum is a rare disease company focused on developing and delivering medicines for conditions where the burden is profound, and the need for new options is significant. Our areas of focus include rare liver and rare genetic diseases, where we are working to advance science for communities that are often overlooked and underserved. Across our work, we aim to bring together science, disciplined development, and close collaboration with patients, families, caregivers, healthcare providers, and advocacy communities.
That commitment has guided Mirum from the beginning. We know that rare disease development requires more than promising science. It requires listening to lived experience, understanding what matters most to families, and designing research programs that reflect the realities of daily life. The voices of patients and caregivers help shape how we think about clinical trials, meaningful outcomes, and the path toward potential future treatment options.
We recognize the strength, knowledge, and persistence of the Fragile X community. Organizations like the National Fragile X Foundation play an essential role in helping families find information, build connections, and advocate for progress. We are grateful for the opportunity to engage with this community and support the continued advancement of research.
The Mirum team recently had the opportunity to attend the National Fragile X Foundation conference in July. Hearing directly from families, researchers, healthcare professionals, and advocates reinforced the strength and dedication that define this community. Our team left inspired by the openness of the conversations, the meaningful connections formed, and the shared commitment to advancing research for people living with Fragile X syndrome.
One of the ways Mirum is contributing to Fragile X research is through the BLOOM clinical study, which is evaluating MRM-3379, an investigational medication, in males aged 13 to 45 diagnosed with Fragile X syndrome. MRM-3379 is being studied to better understand its safety and tolerability and whether it may help manage certain symptoms associated with Fragile X syndrome.
Clinical studies are an important part of evaluating investigational therapies. They help researchers learn more about a potential medicine, including how it is tolerated and whether it may have an effect on symptoms that matter to patients and families. Participation in clinical research is a personal decision, and families should speak with their healthcare provider to understand whether a study may be right for them.
At Mirum, we are inspired by families who continue to push for answers, advocate for their loved ones, and help move research forward. We are committed to working with care and urgency as we continue to learn from the Fragile X community and contribute to the search for potential new treatment options.
To learn more about Mirum’s clinical trials, including the BLOOM study, visit mirumclinicaltrials.com/fragilexstudy.
