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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

2023

Current avenues of gene therapy in Pompe disease.

Current opinion in neurology

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2023

Diaphragm pacing and independent breathing in individuals with severe Pompe disease.

Frontiers in rehabilitation sciences

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2023

Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec

Sleep Medicine

2023

Sensitivity of Pharmacokinetics to Differences in the Particle Size Distribution for Formulations of Locally Acting Mometasone Furoate Suspension-Based Nasal Sprays.

Molecular pharmaceutics

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2022

Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.

Frontiers in neurology

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