Hereditary Spastic Paraplegia Type 50
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls. [Phase 3]
A Phase 3 efficacy trial of IT MELPIDA for hereditary spastic paraplegia type 50 is currently recruiting, using concurrent controls.
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia
A registry and natural history study for early onset hereditary spastic paraplegia is currently recruiting.
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CLINICAL_TRIALS research · 2024-10-08Clinical Trial Update: Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) [Phase 1, Phase 2]
Phase 1/2 trial evaluating Melpida, a recombinant AAV9 gene therapy delivering codon-optimized AP4M1 (hAP4M1opt) for SPG50-related hereditar…