Results 131 to 140 of about 7,353 (158)

Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials. [PDF]

Neurotherapeutics
D'Silva A, Barnes J, Djafar J, Bhattacharya K, Yan J, Mohammad S, Bandodkar S, Johnson A, Tchan M, Miteff C, Elvidge KL, Dale RC, Farrar M.   +12 more
europepmc   +1 more source

Reactions, Adaptation, and Support Needs of Siblings of Children with a Life-Limiting Disease: The Parents' Experiences. [PDF]

Children (Basel)
Vatne TM, Pedersen EO, Lie HC.
europepmc   +1 more source

Characterizing Diagnostic Delays in Metachromatic Leukodystrophy: A Real-World Data Approach. [PDF]

J Inherit Metab Dis
Mohajer A, Sevagamoorthy A, Bean K, Mutua S, Pang F, Adang LA.   +5 more
europepmc   +1 more source

Ex Vivo Gene and Cell Therapy in Hematopoietic Stem Cells. [PDF]

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

Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective. [PDF]

Pediatr Neurol
Gavazzi F, Patel V, Erler JA, Charsar B, Vaia Y, Sevagamoorthy A, Vincent A, Woidill S, Wassmer E, Houlden H, Garcia-Cazorla A, Tonduti D, Wolf NI, Van der Knaap M, Bernard G, Adang LA, Vanderver A.   +16 more
europepmc   +1 more source

A scoping review of stem cell models of leukodystrophies: advances in understanding pathophysiological mechanisms. [PDF]

NPJ Genom Med
Chapleau A, Perrier S, Durcan TM, Bernard G.   +3 more
europepmc   +1 more source

Developing a National Network for Leukodystrophy Research and Care in Canada: The CARELeuko Initiative. [PDF]

Neurol Genet
Chapleau A, Le A, Simo J, Venkateswaran S, Lacaze-Masmonteil T, Piscopo VEC, Gauthier S, Villa Tobón F, Alam SS, Lentini L, Brais B, Ernst C, Mitchell JJ, Vinh DC, Zhou Y, Kennedy TE, Goloff N, Riham B, Chapleau R, Smith B, Greger V, Della Rocca J, Louis LM, Dike A, McIntyre LL, McIntyre DF, Tardif J, Lapointe É, Barnett J, Loignon V, Bardai G, Contant S, Durcan TM, La Piana R, Bernard G, as RareKids-CAN, RARE.Qc, CARELeuko.   +37 more
europepmc   +1 more source

Metachromatic leukodystrophy: Disease spectrum and approaches for treatment [PDF]

Best Practice and Research in Clinical Endocrinology and Metabolism, 2015
Metachromatic leukodystrophy is an inherited lysosomal disorder caused by recessive mutations in ARSA encoding arylsulfatase A. Low activity of arylsulfatase A results in the accumulation of sulfatides in the central and peripheral nervous system leading
Nicole Ingeborg Wolf, Jaap Jan Boelens
exaly   +2 more sources