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Clinical efficacy of gene modification therapy and follow-up observation of multidisciplinary comprehensive management in children with spinal muscular atrophy

Clinical efficacy of gene modification therapy and follow-up observation of multidisciplinary comprehensive management in children with spinal muscular atrophy

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
ChiCTR
Registry ID
ChiCTR2200057395
Enrollment
Unknown
Registered
2022-03-11
Start date
2022-03-01
Completion date
Unknown
Last updated
2023-10-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

spinal muscular atrophy

Interventions

gene modification therapy:gene modification therapy

Sponsors

Beijing Children's Hospital, Capital Medical University
Lead Sponsor

Eligibility

Sex/Gender
All
Age
No minimum to 18 Years

Inclusion criteria

Inclusion criteria: 1.Inpatient or outpatient children in the Department of Neurology, Beijing Children's Hospital, aged <18 years old; 2.Genetically diagnosed patients with 5qSMA; 3.Apply at least one gene-modifying drug.

Exclusion criteria

Exclusion criteria: 1.SMA patients with other genotypes; 2.Have not used gene-modified therapeutic drugs; 3.The patient or guardian does not agree to sign the informed consent.

Design outcomes

Primary

MeasureTime frame
exercise assessment scale;—

Countries

China

Contacts

Public ContactJunlan Lv

Beijing Children's Hospital, Capital Medical University

lujunlan@aliyun.com+86 13910957512

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026