A Registered Cohort Study on SMA

ConditionSpinal Muscular Atrophy

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age1-70

SponsorWan-Jin Chen

About this trial

Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord. This is a registered cohort of spinal muscular atrophy (SMA) type I，II and III in China. This study will provide further insights into the clinical course of SMA including overall survival, demographic characteristics, motor function, respiratory support, feeding and nutritional support, growth and development. The correlation of genotype and phenotype will be conducted.

Eligibility criteria

Qualifiers

Patients with SMA types I, II and III

Asymptomatic SMA carriers

Relatives of SMA patients or carriers

Unrelated healthy controls

Disqualifiers

None

Trial design

Treatments tested in this trial

Not listed

Trial groups

2,000 Participants

are grouped into 6 trial groups

Trial groups

See each trial group below.

Sponsors and collaborators

Wan-Jin Chen

Lead sponsor

First Affiliated Hospital of Fujian Medical University

Sponsor institution

Source ClinicalTrials.gov