- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates
Carmen Leon Astudillo, MD
Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)
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
Active clinical trials
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…
- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates
- Ages
- N/A - N/A
- Sexes
- Male
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…
- Investigator
- Carmen Leon Astudillo
- Status
- Accepting Candidates
- Ages
- 3 Months - 24 Months
- Sexes
- All
My publications
Filter publications
24 publications
2026
Assessing airway clearance dysfunction in Friedreich’s ataxia: A focus on peak cough flow
Journal of Neuromuscular Diseases
Publisher's site2026
Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy
Journal of Neuromuscular Diseases
Publisher's site2025
ATS Pediatric Core Curriculum 2024: The Role of the Pediatric Pulmonologist in the Intensive Care Unit
Pediatric Pulmonology
Publisher's site2025
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
Publisher's site2024
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
Nature Medicine
Publisher's site