A Registered Cohort Study on SMA
Recruiting
Observational Study
Spinal Muscular Atrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 1 Week – 70
- Sex
- Any
- Study type
- Observational
- Participants needed
- 2,000 (estimated)
- Sponsor
- Wan-Jin Chen · Other
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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, …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* 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 Criteria:
\* Participants …
Contacts