PubMed Clinical Queries
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Results for Clinical Studies: Therapy/Broad
5 of 99 results sorted by: Most RecentSee all results in PubMed (99)
Hypomyelination caused by a novel homozygous pathogenic variant in FOLR1: complete clinical and radiological recovery with oral folinic acid therapy and review of the literature.
Orphanet J Rare Dis. 2023. PMID: 37443037 Free PMC article. Review.
Alpha-lipoic acid supplementation corrects pathological alterations in cellular models of pantothenate kinase-associated neurodegeneration with residual PANK2 expression levels.
Orphanet J Rare Dis. 2023. PMID: 37046296 Free PMC article.
Complex Heterozygous Polymerase Gamma Mutation and Cerebral Folate Deficiency in a Child with Refractory Partial Status.
Neurol India. 2022. PMID: 36352670 Free article. No abstract available.
Conservative Iron Chelation for Neuroferritinopathy.
Mov Disord. 2022. PMID: 35996824 Free PMC article.
A GLP1 receptor agonist diabetes drug ameliorates neurodegeneration in a mouse model of infantile neurometabolic disease.
Sci Rep. 2022. PMID: 35970890 Free PMC article.