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The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants | Participants who were prescribed with nusinersen sodium injection in Korea according to local marketing authorization. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nusinersen Sodium Injection | Drug | Administered as specified in the treatment arm. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event. | Up to End of Treatment (2 Years) |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores | Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking. | Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Biogen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pusan Natioanl University Hospital | Busan | South Korea | ||||
| Samsung Changwon Hospital |
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com
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| Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) | The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants are asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability where 0=unable, 1=performed with some assistance, and 2=unaided. The overall score is the sum of the scores for all activities. | Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years) |
| Time to Death | Up to End of Treatment (2 years) |
| Number of Participants with Ventilatory Support | Up to End of Treatment (2 years) |
| Changwon |
| South Korea |
| Chungbuk National University Hospital | Cheongju-si | South Korea |
| Kyungpook National University Hospital | Daegu | South Korea |
| Yeungnam University Hospital | Daegu | South Korea |
| Chungnam National University Hospital | Daejeon | South Korea |
| Chonnam National University Hospital | Gwangju | South Korea |
| Chonbuk National University Hospital | Jeonju | South Korea |
| Severance Hospital | Seoul | 03722 | South Korea |
| Asan Medical Center | Seoul | South Korea |
| Korea university Anam Hospital | Seoul | South Korea |
| Korea University Guro Hospital | Seoul | South Korea |
| Samsung Medical Center | Seoul | South Korea |
| Seoul Natioanl University Hospital | Seoul | South Korea |
| Severance Hospital, Yonsei University Health System | Seoul | South Korea |
| Pusan National University Yangsan Hospital | Yangsan | South Korea |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| C000590926 | nusinersen |
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