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 inj…

Spasticity, MuscleMicrocephalyIntellectual Deficiency+3 more

~Estimated

~$10 – $8,750

Elpida Therapeutics SPCNCT05518188

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biolog…

Hereditary Spastic ParaplegiaSPG47SPG50+8 more

~Estimated

~$10 – $750

Boston Children's HospitalNCT04712812