Last Updated: 09/26/2026
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Found 10 publications
2000-2025: Emerging therapies in primary mitochondrial disease: A structured review, horizon scan, and roadmap for the future.
Journal: Therapeutic advances in rare disease
Published: December 06, 2025
Recommendations for the design of therapeutic trials for neonatal seizures.
Journal: Pediatric research
Published: April 02, 2018
Mortality and Causes of Death in Patients with Sporadic Inclusion Body Myositis: Survey Study Based on the Clinical Experience of Specialists in Australia, Europe and the USA.
Journal: Journal of neuromuscular diseases
Published: November 18, 2016
Regulatory and Ethical Issues in Pediatric Clinical Research: Recommendations From a Panel Discussion.
Journal: Journal of clinical pharmacology
Published: November 04, 2016
Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Journal: Nature chemical biology
Published: March 19, 2016
Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Journal: Nature chemical biology
Published: August 19, 2015
Development of the sporadic inclusion body myositis physical functioning assessment.
Journal: Muscle & nerve
Published: July 14, 2015
SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Journal: Nature chemical biology
Published: December 05, 2014
Treatment of sporadic inclusion body myositis with bimagrumab.
Journal: Neurology
Published: November 09, 2014
Showing 1-10 of 10
Last Updated: 09/26/2026