Last Updated: 09/26/2026
Save publications for later
Sign Up
Not sure about your diagnosis?
Check Your Symptoms
Found 7 publications
Natural history of infantile-onset spinal muscular atrophy.
Journal: Annals of neurology
Published: July 10, 2017
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Journal: The Lancet. Neurology
Published: January 29, 2016
Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.
Journal: Annals of clinical and translational neurology
Published: October 30, 2015
AVI-7288 for Marburg Virus in Nonhuman Primates and Humans.
Journal: The New England journal of medicine
Published: July 23, 2015
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
Journal: Annals of neurology
Published: March 26, 2015
Safety and pharmacokinetic profiles of phosphorodiamidate morpholino oligomers with activity against ebola virus and marburg virus: results of two single-ascending-dose studies.
Journal: Antimicrobial agents and chemotherapy
Published: August 27, 2014
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Journal: Annals of neurology
Published: May 31, 2013
Showing 1-7 of 7
Last Updated: 09/26/2026