Follows people with spinal muscular atrophy in China over time
Observes children and adults with spinal muscular atrophy, tracking how their condition progresses.
Plain-language title generated from the registry record
Registry title: A Registered Cohort Study on SMA
Key facts
- Where
- Fuzhou, ChinaSee the site
Where in China
- Department of Neurology, First Affiliated Hospital Fujian Medical UniversityFuzhou, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Wan-Jin Chen
- Trial code
- NCT04010604
- Record updated
- 23 September 2019
- Original description
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.Read full original descriptionShow less
A Registered Cohort Study on Spinal Muscular Atrophy
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.
Inclusion: Patients with SMA types I, II and III; Asymptomatic SMA carriers; Relatives of SMA patients or carriers; Unrelated healthy controls; Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process.
Exclusion: Participants are unable to comply with trial procedures and visit schedule.