NCT02124616

National Registry for Egyptian Pediatric Neuromuscular Diseases

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 200 (estimated)
• Sponsor: Ain Shams University · Academic / Other
• Sex: All
• Age: 1 Month – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Spinal Muscular Atrophy
• Muscular Dystrophy
• Muscle Diseases
• Myasthenic Syndromes
• Polyneuropathies

Timeline

• Start date: 2014-04-01
• Primary completion: 2020-04-01
• Completion: 2020-12-01
• First posted: 2014-04-28
• Last updated: 2014-04-28

Locations

1 site across 1 country: Egypt