Status:
COMPLETED
Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.
Lead Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
1-2 years
Brief Summary
Spinal muscular atrophy (SMA) is a motor neuron disorder caused by the absence of a functional survival of motor neuron 1, telomeric (SMN1) gene. Type I SMA, a lethal disease of infancy, accounts for ...
Detailed Description
Spinal muscular atrophy (SMA) is a motor neuron disorder caused by the absence of a functional survival of motor neuron 1, telomeric (SMN1) gene. Type I SMA, a lethal disease of infancy, accounts for ...
Eligibility Criteria
Inclusion
- newborn babies
Exclusion
- non-newborn
Key Trial Info
Start Date :
November 11 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
March 30 2021
Estimated Enrollment :
5065 Patients enrolled
Trial Details
Trial ID
NCT05237544
Start Date
November 11 2019
End Date
March 30 2021
Last Update
February 14 2022
Active Locations (1)
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1
Dubowitz Neuromuscular Centre
London, United Kingdom, WC1N 1EH