Fetal Research Affinity Group
Fetal therapy is a rapidly evolving field that involves interventions aimed at improving the health of fetuses diagnosed with a wide range of congenital conditions. Nationwide Children’s Hospital has committed to developing a state-of-the-art fetal therapy program. This undertaking includes not only offering fetal surgeries for life-limiting congenital anomalies but also severe medical conditions not yet amenable to treatment. Within those conditions are several genetic and metabolic syndromes in which organic damage starts well before birth, putting the infant at risk of death or irreversible handicap for life. Current advances in early non-invasive genetic diagnosis, coupled with new developments in enzyme replacement therapies (ERT), gene editing and stem cell research, have made these once inaccessible diseases a renewed target for innovative therapies starting in utero, aiming to improve not only the perinatal but also the long-term outcome.
Our Mission
To bring together experts in fetal therapy, neonatology, genetics, molecular biology and others perinatal fields to develop and advance cutting-edge therapies that can be used to treat fetuses and neonates with a wide range of genetic and medical disorders.
Develop fetal gene therapy, ERT and stem cell transplant for the treatment of conditions such as lysosomal storage diseases, neuromuscular disorders and other inborn errors of metabolism.
Our Goals
- Establish a collaborative research program focused on the development of fetal gene therapy, ERT and stem cell transplant.
- Enhance the understanding of the prenatal phenotype and natural history of this condition using state-of-the-art imaging and laboratory methodologies.
- Develop a referral network aimed at identifying families with an affected child so they can benefit from early diagnosis and therapy for at risk future siblings.
- Develop a database of fetal and neonatal patients with prenatally detected single gene disorders, to assess long-term follow up and outcomes both with and without fetal/neonatal therapies.
- Organize educational events, webinars and workshops to disseminate information about these innovative therapies and to train clinicians and researchers in their use.
- Collaborate with industry partners and funding agencies to secure the necessary resources to support our research activities.
Our Activities
- Monthly virtual and in-person meetings to discuss ongoing research projects, share knowledge and resources and plan future research activities.
- Establishment of collaborative research projects with external research groups and industry partners.
- Organization of educational events and webinars to disseminate information about fetal gene therapy, ERT and stem cell transplant and to train clinicians in their use.
Membership Perks
Inside the Fetal Research Affinity Group
| Date | Discussion Topic | Speaker |
| June | "What Is New in Mucopolysaccharidosis" | Kristen Truxal, MD |
| August | "Fetal Head and Neck MRI" | |
| September | "What Is New in Gene Therapy for Neuromuscular Disorders?" | |
| November | “Advances in the Treatment of Leukodystrophies” |
2024
| Date | Discussion Topic | Speaker |
| February | “Prenatal Therapies for Lysosomal Storage Diseases” | Mari Mori, MD, MS |
2023
| Date | Discussion Topic | Speaker |
| August | Inaugural talk: "Fetal Therapy: Present and Future" |
Adolfo Etchegaray, MD |
| November |
NURAG + FRAG Joint Meeting: "Fetal Therapy update for Lower Urinary Tract Obstruction" |
Adolfo Etchegaray, MD |
| December |
“Exploring Perspectives on Prenatal Therapeutic Interventions in Spinal Muscular Atrophy” |
Afrooz Rashnonejad, MSc, PhD |
