National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
Spinal Muscular Atrophy
|Study Type:||Observational [Patient Registry]|
|Study Design:||Observational Model: Case-Only
Time Perspective: Prospective
|Target Follow-Up Duration:||5 Years|
|Official Title:||National Registry for Egyptian Pediatric Neuromuscular Diseases|
- Functional motor ability [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Motor power in acquired acute neuromuscular diseases will be assessed at admission and morbidity and mortality at discharge from hospital.
Functional motor ability will be performed every 3 months for children with inherited neuromuscular diseases.
- Cardio-pulmonary function [ Time Frame: 12 months ] [ Designated as safety issue: No ]Pulmonary and cardiac function impairment.
|Study Start Date:||April 2014|
|Estimated Study Completion Date:||December 2020|
|Estimated Primary Completion Date:||April 2020 (Final data collection date for primary outcome measure)|
Prospective cohort of children with inherited or acquired neuromuscular diseases.
Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis).
Participants: Eligible infants and children with inherited and acquired neuromuscular diseases.
DESIGN: This study is a prospective cohort study.
Outcome measures: Motor development assessment, respiratory and cardiac examination.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02124616
|Contact: Sahar MA Hassanein, MD, PhDfirstname.lastname@example.org|
|Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University||Recruiting|
|Cairo, Abassia, Egypt, 11381|
|Contact: Mohsen S Elalfy, MD, PhD email@example.com|
|Principal Investigator: Sahar MA Hassanein, MD, PhD|
|Sub-Investigator: Nafissa El Badawy El Badawy, MD, PhD|
|Sub-Investigator: Abdelfattah Souad F Souad, MD, PhD|
|Study Chair:||Sahar MA Hassanein, MD, PhD||Pediatric Department, Children's Hospital, Faculty of Medicine, Ain Shams University|