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(352) 265-8250

Carmen Leon Astudillo, MD

Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)

Photo of Carmen Leon Astudillo
(352) 265-8250 MyUFHealth

Research at a glance

Top areas of exploration

  • Genetic Therapy , 2 publications
  • Sleep Apnea, Obstructive , 2 publications
  • Pediatrics , 2 publications
  • Immunoglobulins, Intravenous , 2 publications

Research activity

19 publications

133 citations

Why is this important?

Active clinical trials

BN44621 Hinalea 2

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a鈥�

Investigator
Carmen Leon Astudillo
Status
Accepting Candidates
Ages
3 Months - 24 Months
Sexes
All
SRP-9001-305 EXPEDITION

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be鈥�

Investigator
Carmen Leon Astudillo
Status
Accepting Candidates
Ages
N/A - N/A
Sexes
Male

My publications

19 publications

2025

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Nature Medicine

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2024

ATS core curriculum 2023. Pediatric pulmonary medicine: Respiratory disorders in infants

Pediatric Pulmonology

2024

Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies

Pediatric Pulmonology

2024

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.

The Journal of clinical investigation

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2023

ATS Core Curriculum 2022. Pediatric Pulmonary Medicine: Updates in pediatric neuromuscular disease

Pediatric Pulmonology