Q3 2021 Investor Relations Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
References
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
CRM
IHD
ZolgensmaⓇ - SMN1 gene replacement therapy
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
Target Patients
NCT03381729 STRONG (COAV101A12102)
Type 2 spinal muscular atrophy
Phase 1
51
Safety and tolerability, incidence of adverse events
Proportion of patients achieving Standing Milestone
Change in Hammersmith Functional Motor Scale
Open-label, single-arm, single-dose, intrathecal
Patients with spinal muscular atrophy with 3 copies of SMN2
Cohort B: Q4-2019 (actual); Cohort C1: TBC
Read-out Milesstone(s)
Publication
TBD
92 Investor Relations | Q3 2021 Results
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