Muscular Atrophy, Spinal
Conditions
Brief summary
The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).
Interventions
None listed
Sponsors
Biogen
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Inclusion criteria
Key Inclusion Criteria: -Genetically confirmed 5q-SMA. Key
Exclusion criteria
-Other types of SMA (non 5q-SMA). NOTE: Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants | Up to 60 Months | The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation. |
| Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants | Up to 60 Months | The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation. |
Countries
China
Contacts
STUDY_DIRECTORMedical Director
Biogen
Outcome results
None listed