Carmen Leon Astudillo, M.D.
Clinical Assistant Professor
About Carmen Leon Astudillo
Carmen Leon Astudillo, MD, is a board-certified pediatric pulmonologist. She received her medical degree from Universidad del Azuay, in Ecuador. After completing her pediatric residency at Cincinnati Children’s Hospital Medical Center, she trained in Pediatric Pulmonology at Boston Children’s Hospital. Dr. Leon Astudillo joined the University of Florida as a clinical assistant professor in 2018. Her clinical and research duties focus mainly on patients with neuromuscular and rare diseases, in particular those with spinal muscular atrophy and Duchenne muscular dystrophy. Dr Leon Astudillo is also part of the aerodigestive program at the University of Florida.
Board Certifications
-
Pediatric PulmonologyAmerican Board of Pediatrics
-
PediatricsAmerican Board of Pediatrics
Clinical Profile
Specialties
- Pediatrics
Subspecialties
- Pediatric Neuromuscular
- Pediatric Pulmonary
Areas of Interest
- Asthma
- Asthma in Children
- Duchenne Muscular Dystrophy
- Friedreich Ataxia
- Myotonia Congenita
- Pompe Disease
- Spinal Muscular Atrophy
Publications
Academic Articles
2025
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
Nature Medicine.
31(1):332-341
[DOI] 10.1038/s41591-024-03304-z.
[PMID] 39385046.
2025
Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy
Journal of Neuromuscular Diseases.
[DOI] 10.1177/22143602251324857.
2024
ATS core curriculum 2023. Pediatric pulmonary medicine: Respiratory disorders in infants
Pediatric Pulmonology.
59(6):1552-1568
[DOI] 10.1002/ppul.26961.
2024
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies
Pediatric Pulmonology.
59(5):1364-1371
[DOI] 10.1002/ppul.26919.
2024
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
The Journal of clinical investigation.
134(1)
[DOI] 10.1172/JCI173510.
[PMID] 37988172.
2024
Twelve Tips to Succeed as Health Profession Clinical Educator in Resource-Limited Settings
Advances in Medical Education and Practice.
Volume 15:201-206
[DOI] 10.2147/amep.s453993.
2023
ATS Core Curriculum 2022. Pediatric Pulmonary Medicine: Updates in pediatric neuromuscular disease
Pediatric Pulmonology.
58(7):1866-1874
[DOI] 10.1002/ppul.26448.
2023
Current avenues of gene therapy in Pompe disease.
Current opinion in neurology.
36(5):464-473
[DOI] 10.1097/WCO.0000000000001187.
[PMID] 37639402.
2023
Diaphragm pacing and independent breathing in individuals with severe Pompe disease.
Frontiers in rehabilitation sciences.
4
[DOI] 10.3389/fresc.2023.1184031.
[PMID] 37583873.
2023
Polysomnography findings in children with spinal muscular atrophy after onasemnogene-abeparvovec
Sleep Medicine.
101:234-237
[DOI] 10.1016/j.sleep.2022.11.006.
2023
Sensitivity of Pharmacokinetics to Differences in the Particle Size Distribution for Formulations of Locally Acting Mometasone Furoate Suspension-Based Nasal Sprays.
Molecular pharmaceutics.
20(11):5690-5700
[DOI] 10.1021/acs.molpharmaceut.3c00553.
[PMID] 37773975.
2022
Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs.
Frontiers in neurology.
13
[DOI] 10.3389/fneur.2022.1064194.
[PMID] 36578307.
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.
30(12):3503-3504
[DOI] 10.1016/j.ymthe.2022.11.007.
[PMID] 36436512.
2021
ATS Core Curriculum 2021. Pediatric Pulmonary Medicine: Pulmonary Infections.
ATS scholar.
2(3):452-467
[DOI] 10.34197/ats-scholar.2021-0034RE.
[PMID] 34667993.
2019
An Unusual Case of Noisy Breathing in an Infant.
Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine.
15(1):149-152
[DOI] 10.5664/jcsm.7590.
[PMID] 30621832.
2019
International Medical Graduates in Pediatrics: Should I Stay or Should I Go?
JAMA pediatrics.
173(3):217-218
[DOI] 10.1001/jamapediatrics.2018.4606.
[PMID] 30667478.
2019
Supplemental Oxygen for Treatment of Infants With Obstructive Sleep Apnea.
Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine.
15(8):1115-1123
[DOI] 10.5664/jcsm.7802.
[PMID] 31482833.
2019
Treatment of severe persistent asthma with IL-6 receptor blockade.
The journal of allergy and clinical immunology. In practice.
7(5):1639-1642.e4
[DOI] 10.1016/j.jaip.2019.02.043.
[PMID] 30885880.
2018
Breath-based diagnosis of fungal infections.
Journal of breath research.
12(2)
[DOI] 10.1088/1752-7163/aa98a1.
[PMID] 29109305.
2018
Performance of International Medical Graduates in Pediatric Residency: A Study of Peer and Faculty Perceptions.
Academic pediatrics.
18(7):728-732
[DOI] 10.1016/j.acap.2018.07.006.
[PMID] 30056222.
2016
Uncovering a Unique Cause of Parotitis in an Adolescent.
Clinical pediatrics.
55(9):880-2
[DOI] 10.1177/0009922815603677.
[PMID] 26324663.
Grants
Sep 2024
ACTIVE
A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY WHO EXPERIENCED A PLATEAU OR DECLINE IN FUNCTION AFTER GENE THERAPY
Role: Principal Investigator
Funding: GENENTECH INC
May 2024
ACTIVE
A PHASE IV OPEN-LABEL STUDY EVALUATING THE EFFECTIVENESS AND SAFETY OF RISDIPLAM ADMINISTERED AS AN EARLY INTERVENTION IN PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY AFTER GENE THERAPY
Role: Principal Investigator
Funding: GENENTECH INC
Feb 2024
ACTIVE
A Phase 3, Multi national, Long-Term Follow-Up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study
Role: Principal Investigator
Funding: SAREPTA THERAPEUTICS
Jul 2023
– Oct 2023
Swallowing Degradation in Untreated Natural History Patients with Spinal Muscular Atrophy Type 1: Establishing an International Registry of Bulbar Dysfunction
Role: Principal Investigator
Funding: UNIV OF MINNESOTA
via GENENTECH INC
Jun 2022
ACTIVE
A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
Role: Principal Investigator
Funding: SAREPTA THERAPEUTICS
Dec 2021
ACTIVE
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects with Duchenne Muscular Dystrophy (EMBARK)
Role: Principal Investigator
Funding: SAREPTA THERAPEUTICS
Jul 2021
ACTIVE
CTOA
Role: Principal Investigator
Funding: UNIV OF FLORIDA
Jul 2012
– Jun 2022
Earl and Christy Powell University Chair in Gene Therapy and Genetics Research – F017885/6
Role: Project Manager
Funding: UF FOUNDATION
Education
Fellowship – Pediatric Pulmonology
2015-2018
·
Boston Children's Hospital
Residency – Pediatrics
2012-2015
·
Cincinnati Children's Hospital Medical Center
M.D.
2004-2010
·
Universidad del Azuay
Contact Details
Phones:
- Business:
- (352) 273-8381
Emails:
- Business:
- cleonastudillo@ufl.edu
Addresses:
- Business Mailing:
-
1600 SW ARCHER RD
PEDIATRIC – PULMONARY DIVISION
GAINESVILLE FL 32610 - Business Street:
-
HD-506
1600 SW ARCHER RD
Gainesville FL 32610