Pediatrics, Pulmonary and Sleep Medicine

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School of Medicine > Clinical Science Departments > Pediatrics > Pediatrics, Pulmonary and Sleep Medicine

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Featured Publications

Chronic Lung Disease-Related Pulmonary Hypertension in Children.

Journal Article Acta Paediatr · January 2026 Full text Link to item Cite

AAV9 gene therapy to target respiratory insufficiency in the D2.mdx mouse model of duchenne muscular dystrophy.

Journal Article Respir Physiol Neurobiol · 2026 Full text Link to item Cite

Predictors of respiratory decline in neuromuscular disorders.

Journal Article Respir Med · January 2026 Full text Link to item Cite

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Featured Grants

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (Start up)

Clinical Trial · Awarded by Pfizer, Inc. · 2025 - 2040

A Phase 3, Multinational, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical study

Clinical Trial · Awarded by Sarepta Therapeutics, Inc. · 2024 - 2031

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy

Clinical Trial · Awarded by Catalyst Pharmaceuticals, Inc. · 2024 - 2029

Respiratory Characterization of DMD Mouse Models

Research · Awarded by Sarepta Therapeutics, Inc. · 2019 - 2027

Novel Adjunctive Therapies for Pompe Disease

Research · Awarded by National Heart, Lung, and Blood Institute · 2024 - 2027

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