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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

23 publications

232 citations

Why is this important?

Active clinical trials

ENDEAVOR

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

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

Investigator
Carmen Leon Astudillo
Status
Accepting Candidates
Ages
3 Months - 24 Months
Sexes
All

My publications

Filter publications

23 publications

2023

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

Frontiers in rehabilitation sciences

PubMed • Publisher's site

2023

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

Pediatric Pulmonology

Publisher's site

2023

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

Sleep Medicine

Publisher's site

2022

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

Frontiers in neurology

PubMed • Publisher's site

2022

Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy

PubMed • Publisher's site