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Results for “Methylmalonic Aciduria and Homocystinuria,Cblc Type”

3 trials

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Showing 3 of 3 results

Testing effectiveness (Phase 2)Study completedNCT01793090
What this trial is testing

EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment

Who this might be right for
Methylmalonic Aciduria and Homocystinuria,Cblc TypeGenetic DiseaseRetinopathy
Bambino Gesù Hospital and Research Institute 30
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100