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At Astellas Gene Therapies, our mission is to develop genetic medicines with the potential to remodel patients’ lives. Myotonic Dystrophy Type 1. As part of our dedication to the patients and households we serve, we are constantly seeking to deepen our understanding of the lived experience of these affected by genetic disorders so as to offer entry to info and resources that may very well be useful to the communities we support. Our Patient Partnerships Team is dedicated to bringing affected person expertise into all elements of our growth programs. 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 commitment, dedication and passion that it takes to ensure that our entire group is doing what's greatest for patients. X-Linked Myotubular Myopathy (XLMTM) is a severe rare, genetic situation that impacts skeletal muscles leading to extreme muscle weakness (hypotonia) and profound respiratory distress, often requiring invasive ventilation help. XLMTM is a monogenic disorder, brought on by pathogenic variants in the MTM1 gene, leading to absent or dysfunctional myotubularin protein. Pompe disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It's attributable to acid alpha-glucosidase (GAA) enzyme deficiency resulting from variants in the GAA gene. Absence or deficiency of GAA ends in accumulation of glycogen in the lysosomes of all cells in the body. Myotonic dystrophy type 1 (DM1) is a uncommon, genetic, neuromuscular disease that affects a number of organ systems with symptoms ranging from myotonia and Nano Earth Labs supplement muscle weakness to cardiac and respiratory dysfunction, excessive sleepiness, and intellectual incapacity. If you are interested to be taught extra concerning the drug improvement course of and clinical trials for gene therapy remedies, please see the "Our Pipeline" web page.