Learn more about our programs
SMA

Obtaining Support and Ongoing Care for Children with SMA after Gene Replacement Therapy

Now Available On Demand

Watch Now

Produced in collaboration with Cure SMA and NORD
Support for this patient/caregiver education program was provided by Novartis Gene Therapies

Parents, caregivers, family members, and patients affected by SMA are invited to watch this 6-part educational program where experts discuss their learnings about treatment responses and the needs of those who received gene replacement therapy.

The multidisciplinary panel comprised of clinicians, advocates, and parents of children with SMA will bring to light unique perspectives regarding the needs of patients with SMA, and their families.

The following learning objectives are discussed:

  • Module 1: Understanding Gene Replacement Therapy in SMA
    Discuss the benefits, administration, and treatment outcomes of gene replacement therapy for SMA
  • Module 2: Life After SMA Gene Replacement Therapy
    Describe the beneficial effects of treatment with gene replacement therapy on patients with SMA
  • Module 3: Respiratory Care and SMA
    Identify strategies and/or methods to maintain respiratory health in patients with SMA
  • Module 4: Rehabilitative Care and SMA
    Identify strategies and/or methods to maintain motor function and mobility in patients with SMA
  • Module 5: Nutrition and SMA
    Recognize the therapeutic importance of maintaining optimal nutrition in patients with SMA
  • Module 6: Shared Stories of SMA Families: The Good, Bad, and In-between
    Communicate with other patients and families about the impact SMA has on quality of life, challenges, and best practices

See below to select a module and start watching today!

 

Modules

Speakers

Mary Schroth, MD

Chief Medical Officer, Cure SMA

Mary Schroth, MD, is the chief medical officer of Cure SMA. She is professor emeritus at the University of Wisconsin School of Medicine and Public Health in Madison where she served as a pediatric pulmonologist, co-director of the Pediatric Neuromuscular Clinic, and director of the Pediatric Pulmonary Center Grant. Dr Schroth feels honored to be able to share her expertise of SMA care through her role with Cure SMA.

Julie Parsons, MD

Professor of Clinical Pediatrics and Neurology, Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders. Children's Hospital Colorado, University of Colorado School of Medicine

Julie A. Parsons, MD, is a professor of clinical pediatrics and neurology at the University of Colorado School of Medicine in Aurora, the Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, and co-director of the neuromuscular clinic at Children’s Hospital Colorado. Her clinical interests include neuromuscular diseases such as muscular dystrophies, spinal muscular atrophy (SMA), and myotonic dystrophies. She is an active participant and principal investigator for many clinical trials, and is a member of organizations that include the Child Neurology Society and the American Academy of Neurology. Dr Parsons earned her MD from the University of Colorado School of Medicine, followed by residencies in pediatrics and child neurology and a neuromuscular medicine fellowship.

Richard Shell, MD

Professor of Pediatrics, The Ohio State University Section Chief of Pulmonary Medicine, Nationwide Children's Hospital

Richard Shell, MD, is professor of pediatrics and section chief of the Department of Pulmonary Medicine at Nationwide Children's Hospital at The Ohio State University College of Medicine in Columbus. He plays an active clinical and research role in the care of patients with neuromuscular disease. Dr Shell’s other research interests include asthma and cystic fibrosis, where he assists in many clinical trials within his department. He is a member of the Therapeutic Development Network of the Cystic Fibrosis Foundation and has administrative rolls with both Parent Project Muscular Dystrophy (PPMD) and CureSMA. Dr Shell has been recognized by his patients for excellence in family-centered care. He received his medical degree from the Medical College of Ohio in Toledo.

Linda Lowes, PT, PhD

Associate Professor, Department of Pediatrics. The Ohio State University

Linda P. Lowes, PT, PhD, is associate professor of pediatrics at The Ohio State University and a principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus. Her research interests focus on optimizing functional outcomes in adults and children with neuromuscular disorders. Dr Lowes is also interested in promoting clinical trial readiness through data to drive outcome measure selection. To facilitate clinical trial readiness, she has conducted numerous natural history studies in limb-girdle muscular dystrophies and other disorders. Dr Lowes was recently elected to the executive board for TreatNMD and awarded the Excellence in Innovation award.

Becky Hurst Davis, MS, RD, CSP, CD, CNSC

Nutrition Subcommittee Chair, Pediatric Nutritionist at Intermountain Healthcare. Salt Lake City, UT

Becky Hurst Davis MS, RD, CSP, CD, CNSC, is a registered dietitian working with Intermountain Health Care in Salt Lake City, Utah. She is a certified specialist in pediatric nutrition and a certified nutrition support clinician. Becky has great interest in clinical research providing improved nutrition care to people with neuromuscular diseases and has been involved with Cure SMA for over 11 years. She graduated with her Master of Science in Nutrition from the University of Utah in Salt Lake City.

Sapna Pringle

Caregiver

Sapna Pringle is a licensed CPA and has spent 19 years in public accounting in Houston, Texas, where she lives with her husband and three children. Her oldest daughter, Maya (12), was diagnosed with Spinal Muscular Atrophy (SMA) Type 2 at the age of one. Sapna has been involved with media/speaking engagements and community events on behalf of Muscular Dystrophy Association. She is also active with Cure SMA, where her family concentrates most of their fundraising and awareness efforts. Sapna graduated from Texas A&M University in College Station with a bachelor’s and a master’s degree in accounting.

Amy Medina

Caregiver

Amy Medina is a CPS social worker for Fond du Lac County and lives with her husband, Adan, and their three children, Mateo, Javier, and Amelia, in Wisconsin. All three of their children have Spinal Muscular Atrophy (SMA), and their two youngest have participated in clinical drug trials. Amy serves on the Cure SMA board. She is very passionate on educating others on SMA as well as being a resource and support for the SMA community.

Amanda DeVay

Caregiver

Amanda DeVay is a full-time mom and care provider to her two sons. Her younger son, who is 8 months old, was born with Spinal Muscular Atrophy (SMA), and her older son is non-affected. Since her son’s diagnosis, Amanda has become an advocate for SMA awareness and the importance of early detection through state-mandated newborn screening. She recently spoke as a panelist for the newly diagnosed segment at Cure SMA’s 2020 Annual SMA Conference. Amanda’s background is in account management and customer service relations

 

Partners and Supporters

  • Cure SMA
  • NORD