NCT04010604
A Registered Cohort Study on SMA
Recruiting · Not specified · Wan-Jin Chen · registry updated 2019-09-23
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
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