You are not alone on this journey. Connecting with other families and caregivers who understand what you’re going through can give you emotional support and helpful advice. This section is here to help you find valuable resources, connect with communities, and learn about new research that can support you and your entire family.
Many groups are here to help families affected by XLMTM and other related muscle conditions:
These groups often have helpful information, events, and ways for you to connect with other families. Taking care of your own well-being is essential in the process of caring for loved ones with XLMTM. Look for resources that support your well-being, too.
Researchers are looking for ways to potentially treat XLMTM with investigational gene therapy. The goal of gene therapy is to deliver a healthy copy of a faulty gene to help treat or improve a disease.
You can learn more about research opportunities, including clinical trials on sites like clinicaltrials.gov, and patient registries like the MTM/CNM Registry, designed to further understanding of XLMTM. These efforts help deepen knowledge about the disease, foster connections among families, and empower informed choices.
Astellas believes it’s important to listen to patients and families when developing medicines. We are committed to including the patient voice at every stage of the treatment development life cycle, bringing insights from patient representatives and advocacy organizations into the design and implementation of our studies. This helps ensure that the medicines we develop truly reflect what matters to those who are living with, or impacted by, a disease.
We are pleased to be working with a panel of patient organizations from around the world, including those based in Europe, Latin America, and the United States, to seek their input and insights throughout the research and development process. We look forward to continuing this collaborative work as we move forward.