Treatment with Ataluren for Duchene Muscular Dystrophy

Authors

  • Sameh S. Morkous Lehigh Valley Health Network, Lehigh Valley Reilly Children's Hospital

DOI:

https://doi.org/10.15844/pedneurbriefs-34-12

Keywords:

Pediatric, neuromuscular, Duchenne muscular dystrophy, STRIDE Registry

Abstract

Investigators from Europe and the USA, representing the STRIDE Registry and Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), examined the effectiveness of ataluren and standard of care in the Registry versus stand of care alone in the CINRG DNHS.

 

Author Biography

Sameh S. Morkous, Lehigh Valley Health Network, Lehigh Valley Reilly Children's Hospital

I have been fellowship trained in nationally ranked programs and a Board Certified Child Neurologist with additional fellowship training and Board Certification in Sleep Medicine and also certified in Epilepsy and Headache Medicine. I have a strong experience in outpatient, inpatient, administration, executive and philanthropic work. I did national and international presentations and I am also an experienced medical expert consultant. My scholarly work includes Institutional Review Board (IRB) Research; Peer Reviewed Publications in top tier medical journals; Quality Peer Reviewer for prestigious medical journals, medical associations and professional societies; American Academy of Sleep Medicine (AASM) Political Action Committee (PAC) Northeastern Region (US States) Ambassador; American Academy of Neurology (AAN) peer committee selected advocate including Neurology on the Hill and Palatucci Advocacy Leadership Forum. My background includes dual administrative experience in both Child Neurology and Sleep Medicine. I am motivated by opportunities and challenges to significantly contribute and impact the medical field while building lasting peer and patient relationship

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Published

2020-12-04

Issue

Vol. 34 (2020)

Section

Neuromuscular Disorders

License

Copyright (c) 2020 The Author(s)

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