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Carmen Leon Astudillo, MD

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

Photo of Carmen Leon Astudillo

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

BN44620

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have鈥�

Investigator
Carmen Leon Astudillo
Status
Accepting Candidates
Ages
3 Months - 24 Months
Sexes
All
9001-303 (ENVISION)

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the鈥�

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

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with鈥�

Investigator
Carmen Leon Astudillo
Status
Accepting Candidates
Ages
4 Years - 7 Years
Sexes
Male

My publications

19 publications

2025

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

Nature Medicine

鈥�

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

鈥�

2023

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

Pediatric Pulmonology