National Registry for Egyptian Pediatric Neuromuscular Diseases

Ain Shams University

Status: Unknown

Conditions: Polyneuropathies; Myasthenic Syndromes; Muscle Diseases; Muscular Dystrophy; Spinal Muscular Atrophy

Study type: Observational

Funder types: Other

Identifiers

NCT02124616

EGYPT PED-NMD 2014

Details and patient eligibility

About

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.

Full 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.

Enrollment: 200 estimated patients

Sex: All

Ages: 1 month to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Weakness, hypotonia.
• Nerve conduction study and electromyographic confirmation of lower motor neuron affection.

Exclusion criteria

• Chromosomal diseases.
• Malformations and deformations.

Trial design

200 participants in 1 patient group

Neuromuscular diseases

Description:

Prospective cohort of children with inherited or acquired neuromuscular diseases.