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MechanismAP4M1 stimulants |
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Inactive Indication- |
Drug Highest PhaseClinical |
First Approval Ctry. / Loc.- |
First Approval Date- |
A Phase 1/2 Open-label Intrathecal Administration of MELPIDA to Determine Its Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (SPG50) Caused by Mutation in the AP4M1 Gene.
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Secondarily, the trial will explore efficacy in terms of disease burden assessments.
100 Clinical Results associated with CureSPG50
0 Patents (Medical) associated with CureSPG50
100 Deals associated with CureSPG50
100 Translational Medicine associated with CureSPG50