spinal muscular atrophy
Conditions
Interventions
Case group:Nocinacin sodium intrathecal injection
Sponsors
West China Hospital, Sichuan University
Eligibility
Sex/Gender
All
Age
14 Years to 50 Years
Inclusion criteria
Inclusion criteria: (1) Genetic diagnosis of 5q-SMA. (2) Age =18 at the time of enrollment. (3) If appropriate and applicable, the subject and/or their legally authorized representative (e.g., caregiver) are able to understand the purpose and risks of the study, sign informed consent, and authorize the use of confidential health information in accordance with state and local privacy regulations.
Exclusion criteria
Exclusion criteria: (1) Complicated with serious diseases of the heart, liver, kidney, digestive tract, endocrine, immune system and nervous system; (2) Combined with blood system diseases; (3) Combined with malignant tumor; (4) Combined with serious mental illness: including but not limited to severe depression, suicidal tendencies, bipolar disorder, schizophrenia, etc.; (5) Combined acute/chronic infection; (6) Other types of SMA (not 5q-SMA); (7) Unable or unwilling to sign informed consent; (8) Participating in other interventional clinical trials; (9) Patients judged by the investigator to be unsuitable for clinical trials.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| myodynamia; | — |
Secondary
| Measure | Time frame |
|---|---|
| scoliosis;motor functions;Electrophysiology and biomarkers; | — |
Countries
China
Contacts
Public ContactXiaohui Lai
West China Hospital of Sichuan University
Outcome results
None listed