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The FBRI VTC Neuromotor Research Clinic
The FBRI VTC Neuromotor Research Clinic was established and opened in May of 2013 to provide intensive therapeutic services to individuals with motor impairmen…
Neuromotor ImpairmentsCerebral PalsyMicrocephaly+4 more
Virginia Polytechnic Institute and State UniversityNCT03325946
Phase 1
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 inj…
Spasticity, MuscleMicrocephalyIntellectual Deficiency+3 more
Elpida Therapeutics SPCNCT05518188
Investigations of Individuals With MEHMO Syndrome or eIF2-Pathway Related Conditions
This observational natural history study will follow individuals with MEHMO (Mental disability, Epileptic seizure, Hypopituitarism/Hypogenitalism, Microcephaly…
Intellectual DisabilityEpilepsyHypogonadisms+3 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NCT06019182