Genetics of DMD and Therapeutic Implications
Produced in partnership with NORD and Parent Project Muscular Dystrophy.
This activity is supported by educational grants from PTC Therapeutics, Inc. and Sarepta Therapeutics, Inc.
With the development of disease-modifying therapies (DMTs) and advances in multidisciplinary care, early diagnosis of Duchenne muscular dystrophy (Duchenne) is essential to improving outcomes. With enhanced quality of care, a larger number of patients can now survive into adulthood.
Join us for a new live webinar on the latest developments in the management of Duchenne with a panel of expert clinicians, caregivers, and a representative from Parent Project Muscular Dystrophy (PPMD).
Tune in to learn more about:
- The diagnosis of Duchenne and the importance of early identification
- Newer treatment options for Duchenne
- Functional outcomes of current Duchenne therapies
- The transition from pediatric to adult care
- Identifying key components of multidisciplinary care to optimize outcomes
The Changing Landscape of DMD Management: The Caregiver's Perspective
Addressing the Evolving Needs of the DMD Patient
Preparing for a Future with DMD
John Brandsema, MD
Neuromuscular Section Head
The Children's Hospital of Philadelphia Associate Professor of Clinical Neurology Perelman School of Medicine University of Pennsylvania
John Brandsema, MD, is a child neurologist and the Neuromuscular Section Head at the Children’s Hospital of Philadelphia. He has broad clinical and research interests in neuromuscular diseases, with a particular focus in clinical trials of novel therapies for childhood neuromuscular disorders and is enthusiastic about the promise that medical and scientific advances hold for people with these conditions. Dr. Brandsema is involved with clinical trials of novel therapeutics for neuromuscular disorders. His philosophy of care centers around developing strong partnerships with families and empowering parents to understand their child’s medical condition. Outside of the hospital, he is a classically-trained singer and musician and a budding Philadelphia sports fan.
Melissa Gibbons, MS, CGC
Certified Genetic Counselor
Assistant Professor University of Colorado School of Medicine Children's Hospital Colorado
Melissa Gibbons, MS, is an assistant professor at University of Colorado School of Medicine and a certified genetic counselor at Children’s Hospital Colorado. Melissa has been a genetic counselor in the Multidisciplinary Neuromuscular Clinic at Children’s Hospital Colorado for 15 years. She has been involved in clinical care and clinical research for SMA and DMD. Her clinical interests include neurogenetics, neuromuscular and spinal muscular atrophy newborn screening. In addition, Melissa is the SMA newborn screen coordinator for Colorado and Wyoming and a member of CureSMA Medical Advisory Committee. Furthermore, she has received the Paul G Moe, MD Excellence in Child Neurology Award, Section of Neurology in 2018 for exemplary service in teaching, teamwork and patient care.
Alexis Hazlett, MSN, RN, CPN
Director of Family Support & Case Management
Parent Project Muscular Dystrophy (PPMD)
Alexis Hazlett, MSN, RN, CPN is Director of Family Support and Case Management at Parent Project Muscular Dystrophy (PPMD) located in Washington, DC. Ms. Hazlett is from Kansas City, Missouri and previously served as the neuromuscular nurse coordinator at Children’s Mercy Hospital, Kansas City before joining Parent Project Muscular Dystrophy in 2022. Ms. Hazlett’s role is multifaceted and serves to provide direct-to-family support and case management services. Ms. Hazlett has an interest in health equity, accessibility, access to care, and health literacy.
Gretchen Agans, MSW Candidate
Mother of a teenage boy living with Duchenne
Ethics and Advance Care Planning Researcher
Gretchen Agans is an Advance Care Planning researcher within the Ethics Department at Wellstar Health System in Atlanta, Georgia. To better serve the community, she is expanding her knowledge and credentials as a Master of Social Work candidate at Columbia University. Her goals include earning a PhD in social work while formally investigating the various pathways to caregiver thriving, particularly within the end-of-life space. Gretchen is the mother of five children, one of whom is living with Duchenne Muscular Dystrophy. He is 15 years old and non-ambulatory.