Results 91 to 100 of about 13,975 (131)

Late-Onset X-linked Adrenoleukodystrophy: A Rare Cause of Progressive Spastic Paraparesis. [PDF]

Cureus
Sequeira S, Costa A, Vargas M, Velon A.
europepmc   +1 more source

Living with adrenoleukodystrophy: adult patient and caregiver perspectives. [PDF]

Orphanet J Rare Dis
Fine AS, Sullivan-Fortin KO, Miettunen K, Emerson F, Sadjadi R, Fatemi A, Eichler F.   +6 more
europepmc   +1 more source

Oncogenesis as an Adverse Effect of Gene Replacement Therapy in Hematopoietic Stem Cells. [PDF]

Int J Mol Sci
Petrova IO, Smirnikhina SA.
europepmc   +1 more source

CSF and plasma neuro-biomarker dynamics after gene therapy for cerebral adrenoleukodystrophy. [PDF]

Mol Ther Adv
Lund TC, Gupta AO, Kemp S, Loes DJ, Nascene DR, Orchard PJ.   +5 more
europepmc   +1 more source

Neuroregenerative Treatment With Radio Electric Asymmetric Conveyer Technology in Advanced Childhood Cerebral X-linked Adrenoleukodystrophy: Clinical Stabilization and Electroencephalographic Evidence of Cortico-Subcortical Reorganization. [PDF]

Cureus
Fontani V, Rinaldi A, Rinaldi S.
europepmc   +1 more source

Recognizing Cerebral Pathology in Adrenoleukodystrophy Amid the Noise. [PDF]

Neurology
Pierpont EI.
europepmc   +1 more source

Natural History of Clinical Phenotypes and Their Biochemical Correlates in Adult X-Linked Adrenoleukodystrophy. [PDF]

J Inherit Metab Dis
Lier J, Ponleitner M, Popp D, Schäfer L, Küstermann F, Meigen C, Kratzer F, Janda J, Okun J, Jamra RA, Köhler W, Bergner C.   +11 more
europepmc   +1 more source

Sexual Dysfunction and Its Relationship With Hypogonadism and Myelopathy in Male Patients With X-Linked Adrenoleukodystrophy. [PDF]

J Inherit Metab Dis
van de Stadt SIW, Wessel AMA, Langeveld M, Engelen M, Sjouke B.   +4 more
europepmc   +1 more source

An Automated Analysis Tool for Diffusion Tensor Imaging-Based Quantitative MRI in X-Linked Adrenoleukodystrophy. [PDF]

J Inherit Metab Dis
Kabak EG, van Dodewaard F, Roosendaal SD, Yska HAF, Voermans MMC, Bourgeais M, Perlbarg V, Engelen M.   +7 more
europepmc   +1 more source

A comprehensive discovery platform for ELOVL1 small-molecule inhibitors targeting very long-chain fatty acid synthesis in adrenoleukodystrophy. [PDF]

J Biol Chem
Holley S, Asmussen G, Lam B, Zhao Z, Freed B, Guo L, Dostalova Z, Tang B, Kothe M, Lang P, Wang D, Hanus M, Belenky A, Cromwell M, Kloss A, Gladysheva T.   +15 more
europepmc   +1 more source