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At Astellas Gene Therapies, our mission is to develop genetic medicines with the potential to transform patients’ lives. Myotonic Dystrophy Type 1. As part of our commitment to the patients and households we serve, we're continually seeking to deepen our understanding of the lived experience of those affected by genetic disorders in order to provide entry to information and resources that could possibly be useful to the communities we support. Our Patient Partnerships Team is dedicated to bringing affected person experience into all aspects of our development packages. Our precedence is to weave affected person and caregiver perspectives into the fabric of all that we do on a day-to-day foundation. And we advocate for patients and families with the dedication, dedication and passion that it takes to ensure that our whole group is doing what is finest for patients. X-Linked Myotubular Myopathy (XLMTM) is a critical uncommon, genetic condition that affects skeletal muscles leading to extreme muscle weakness (hypotonia) and profound respiratory distress, typically requiring invasive ventilation help. XLMTM is a monogenic disorder, attributable to pathogenic variants in the MTM1 gene, resulting in absent or dysfunctional myotubularin protein. Pompe disease is a uncommon, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It's brought on by acid alpha-glucosidase (GAA) enzyme deficiency ensuing from variants within the GAA gene. Absence or deficiency of GAA leads to accumulation of glycogen in the lysosomes of all cells within the body. Myotonic dystrophy sort 1 (DM1) is a uncommon, genetic, neuromuscular illness that affects a number of organ techniques with symptoms starting from myotonia and muscle weakness to cardiac and respiratory dysfunction, excessive sleepiness, and mental incapacity. If you are interested to be taught extra about the drug growth process and clinical trials for gene therapy therapies, please see the "Our Pipeline" web page.