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Novel Therapies for Genetic and Rare Pediatric Diseases

Rare and genetic diseases often pose diagnostic and therapeutic challenges, but novel therapies for genetic and rare pediatric diseases are offering renewed hope. Gene therapy, enzyme replacement, antisense oligonucleotides, and mRNA-based interventions are gaining traction for conditions once considered untreatable. Diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and certain metabolic disorders have seen transformative progress with recently approved therapies. Personalized treatment plans guided by molecular diagnostics improve quality of life and long-term prognosis. Multidisciplinary teams work closely with genetic counselors and regulatory bodies to ensure responsible access. As the pipeline for rare disease therapies expands, attention to affordability and global accessibility will be vital to equitable care.

Committee Members
Speaker at Pediatrics and Neonatology 2025 - Steven M Donn

Steven M Donn

University of Michigan, United States
Speaker at Pediatrics and Neonatology 2025 - Peter Averkiou

Peter Averkiou

Florida Atlantic University, United States
Speaker at Pediatrics and Neonatology 2025 - Catherine Fallet Bianco

Catherine Fallet Bianco

Sainte-Justine University Hospital, Canada
Speaker at Pediatrics and Neonatology 2025 - Jeanne Magagna

Jeanne Magagna

Great Ormond Street Hospital for Children, United Kingdom
EPN 2025 Speakers
Speaker at Pediatrics and Neonatology 2025 - Ann L Smith

Ann L Smith

Novant Health Forsyth Medical Center, United States
Speaker at Pediatrics and Neonatology 2025 - Tehreem Fatima

Tehreem Fatima

University of Connecticut, United States
Speaker at Pediatrics and Neonatology 2025 - Rohit Kumar

Rohit Kumar

James Cook Hospital, United Kingdom
Speaker at Pediatrics and Neonatology 2025 - Nicola Webster

Nicola Webster

North West Regional Hospital, Australia
Speaker at Pediatrics and Neonatology 2025 - Tadel Karolina

Tadel Karolina

Wroclaw Medical University, Poland
Speaker at Pediatrics and Neonatology 2025 - Vlad Dima

Vlad Dima

Filantropia Clinical Hospital, Romania
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