This episode is in partnership with Boston Biotechnology Summit, a bridge to collaboration and innovative synergies between healthcare stakeholders. In this conversation, we speak with Matthias Lambert, a scientist and patient advocate, who shares his remarkable journey from being a patient to becoming a scientist. Matthias was born with a rare neuromuscular disease. Despite being the only one in his family with this condition, he didn't know the genetic cause for 25 years. However, during his PhD, he discovered the gene responsible for his disease, TPM3. Matthias now works at a lab in Boston, focusing on neuromuscular diseases. He emphasizes the importance of patient scientists in drug discovery, highlighting that their work directly impacts their lives and the lives of others with similar conditions. Matthias discusses the challenges of working on rare diseases, including funding and the scarcity of research talent in the field. He explains the significance of innovation in gene therapy, particularly in finding effective delivery methods to target specific tissues. Matthias stresses the need for a more inclusive approach to research, with patients playing a crucial role in decision-making processes. Lastly, Matthias urges healthcare leaders to listen to patients, emphasizing that patients often know more about their own diseases than anyone else. He encourages patients to speak up and pursue their ideas, serving as an inspiration and beacon of hope for others facing similar challenges. "Our metric is to save lives" MEET OUR GUEST Matthias Lambert Senior Scientist in the division of Genetics and Genomics at Boston Children's Hospital. Matthias Lambert is a Senior Scientist in the division of Genetics and Genomics at Boston Children's Hospital, and Instructor in the department of Pediatrics at Harvard Medical School. Matthias identifies as a patient-scientist, navigating life with a rare congenital myopathy. His research focuses on studying the mechanisms that contribute to the pathogenesis of rare muscle diseases including his own genetic condition. He is actively engaged in pioneering efforts to advance cutting-edge therapies for these conditions. His work is supported by prestigious grants from the National Institutes of Health, the Muscular Dystrophy Association (MDA), and the AFM-Telethon. In 2024, Matthias plans to establish his own independent research group.