We are dedicating this episode to ‘Women & Duchenne’ in honor of World Duchenne Awareness Day which takes place every September 7. Tune in to hear from three incredible women: a clinical expert, a mother who is also a carrier and caregiver, and a scientist share their knowledge, perspectives and experiences. Each has a set of unique challenges to overcome in order to manage Duchenne Muscular Dystrophy (DMD).
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A professor of pediatrics and a pediatric cardiologist for The Heart Center. She is also a member of the physician team for the Neuromuscular Disorders section of The Neurosciences Center at Nationwide Children’s Hospital. Dr. Cripe completed her residency at the University of Iowa Hospitals and Clinics. She served as a pediatric cardiology fellow at the University of Iowa Hospitals and Clinics, and at Children’s Hospital Boston. Before coming to Nationwide Children’s, Dr. Cripe spent 12 years at Cincinnati Children’s Hospital Medical Center. Dr. Cripe’s clinical interests focus on non-invasive cardiac imaging specifically echocardiography as well as on the care and treatment of cardiomyopathy associated with neuromuscular disease, such as Duchenne muscular dystrophy. She was a member of the Centers for Disease Control (CDC) National Steering Committee Duchenne Muscular Dystrophy Standards of Care, and has been an invited lecturer nationally and internationally on cardiomyopathy related to DMD. Dr. Cripe is currently serving as a member of the Scientific Advisory Board for Parent Project Muscular Dystrophy.
A military spouse and mother of four young children, Elizabeth’s journey with DMD began when her son was diagnosed at the age of five. Elizabeth shares the importance of support and accessibility for all those touched by DMD, while also raising awareness for the disease and emphasizing the importance of surveillance and treatment for carriers like herself and her daughter.
Ellen Welch has applied her understanding of post-transcriptional control mechanisms to the discovery and development of small molecule therapeutics that modulate RNA-mediated processes.
Over the last 20 years, she has been involved in the discovery and development of small molecules that promote the readthrough of nonsense codons and in the identification of small molecules that modulate splicing. The application of these mechanisms and others are the focus of her ongoing efforts to bring therapies to patients with rare disorders.
Ellen earned her doctorate of philosophy in molecular biology from the University of Massachusetts Medical School and completed her postdoctoral studies in molecular biology at Robert Wood Johnson Medical School at Rutgers University.
Jonathan Blaize received his PhD in Neuroscience from the City University of New York before joining the faculty at Wagner College where he earned tenure and rank of Associate Professor. A learner by nature, Jon studied the molecular underpinnings of vision and neural inhibition, epigenetic remodeling, and phthalate driven endocrine disruption during his academic tenue. Jon taught more than 20 different classes, developed new courses and designed curricula while also serving as Co-Director of Pre-Professional Programs, Physician’s Assistant Advisor, Grants Coordinator, and curriculum assessment consultant, to name a few. Having focused on translational research throughout his career, and with a desire to engage the rare disease community while leveraging his vast experience in education, Jon pursued board certification in Medical Affairs and transitioned to the role of Medical Science Liaison at PTC Therapeutics in December of 2021. Jon remains passionate about patient care, disease state awareness, education access and research advancement and is thrilled to provide a platform for patients, care providers and scientists to share their experiences and knowledge on the Take-On Duchenne North American Podcast.
US-DMD-0294 I November 2022
PTC Therapeutics has provided financial compensation to
Elizabeth Floyd and Dr. Cripe for the time to conduct this interview.
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