Prospective Observational Study on the Method to Assess the Efficacy of Risdiplam on Manual Dexterity in Patients with Spinal Muscular Atrophy
- Conditions
- Spinal Muscular Atrophy
- Registration Number
- JPRN-UMIN000045717
- Lead Sponsor
- CHUGAI PHARMACEUTICAL CO.LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 30
Not provided
<Cohort A> (1) Patients who have received Nusinersen <Cohort A and Cohort B> (1) Patients who have received Risdiplam <Cohort A, Cohort B and Cohort C> (1) Patients who have received Onasemnogene abeparvovecor other SMN2 modifiers (2) Patients who are receiving valproic acid. (3) Patients who have undergone surgery for scoliosis or hip arthrodesis within 6 months before screening or are scheduled to undergo within 18 months after screening (4) Patients who require invasive ventilation or tracheostomy (5) Patients who are judged by the investigator to have a disease other than SMA that causes functional decline in the upper limbs (6) Patients who are judged by the investigator not to be suitable for this study for any reason
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method