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6. Detailed Clinical Phenotype and Molecular Genetic Findings in CLN3-Associated Isolated Retinal Degeneration. Ku CA; Hull S; Arno G; Vincent A; Carss K; Kayton R; Weeks D; Anderson GW; Geraets R; Parker C; Pearce DA; Michaelides M; MacLaren RE; Robson AG; Holder GE; Heon E; Raymond FL; Moore AT; Webster AR; Pennesi ME JAMA Ophthalmol; 2017 Jul; 135(7):749-760. PubMed ID: 28542676 [TBL] [Abstract][Full Text] [Related]
7. Autophagic vacuolar myopathy is a common feature of CLN3 disease. Radke J; Koll R; Gill E; Wiese L; Schulz A; Kohlschütter A; Schuelke M; Hagel C; Stenzel W; Goebel HH Ann Clin Transl Neurol; 2018 Nov; 5(11):1385-1393. PubMed ID: 30480032 [TBL] [Abstract][Full Text] [Related]
8. Late adult-onset of X-linked myopathy with excessive autophagy. Crockett CD; Ruggieri A; Gujrati M; Zallek CM; Ramachandran N; Minassian BA; Moore SA Muscle Nerve; 2014 Jul; 50(1):138-44. PubMed ID: 24488655 [TBL] [Abstract][Full Text] [Related]
9. Clinical and molecular characterization of non-syndromic retinal dystrophy due to c.175G>A mutation in ceroid lipofuscinosis neuronal 3 (CLN3). Chen FK; Zhang X; Eintracht J; Zhang D; Arunachalam S; Thompson JA; Chelva E; Mallon D; Chen SC; McLaren T; Lamey T; De Roach J; McLenachan S Doc Ophthalmol; 2019 Feb; 138(1):55-70. PubMed ID: 30446867 [TBL] [Abstract][Full Text] [Related]
10. A novel form of autophagic vacuolar myopathy with late-onset and multiorgan involvement. Kaneda D; Sugie K; Yamamoto A; Matsumoto H; Kato T; Nonaka I; Nishino I Neurology; 2003 Jul; 61(1):128-31. PubMed ID: 12847175 [TBL] [Abstract][Full Text] [Related]
11. Autophagic vacuolar myopathy in twin girls. Holton JL; Beesley C; Jackson M; Venner K; Bhardwaj N; Winchester B; Al-Memar A Neuropathol Appl Neurobiol; 2006 Jun; 32(3):253-9. PubMed ID: 16640643 [TBL] [Abstract][Full Text] [Related]
12. Next generation sequencing-based molecular diagnosis of retinitis pigmentosa: identification of a novel genotype-phenotype correlation and clinical refinements. Wang F; Wang H; Tuan HF; Nguyen DH; Sun V; Keser V; Bowne SJ; Sullivan LS; Luo H; Zhao L; Wang X; Zaneveld JE; Salvo JS; Siddiqui S; Mao L; Wheaton DK; Birch DG; Branham KE; Heckenlively JR; Wen C; Flagg K; Ferreyra H; Pei J; Khan A; Ren H; Wang K; Lopez I; Qamar R; Zenteno JC; Ayala-Ramirez R; Buentello-Volante B; Fu Q; Simpson DA; Li Y; Sui R; Silvestri G; Daiger SP; Koenekoop RK; Zhang K; Chen R Hum Genet; 2014 Mar; 133(3):331-45. PubMed ID: 24154662 [TBL] [Abstract][Full Text] [Related]
13. A new congenital form of X-linked autophagic vacuolar myopathy. Yan C; Tanaka M; Sugie K; Nobutoki T; Woo M; Murase N; Higuchi Y; Noguchi S; Nonaka I; Hayashi YK; Nishino I Neurology; 2005 Oct; 65(7):1132-4. PubMed ID: 16217076 [TBL] [Abstract][Full Text] [Related]
14. Asymptomatic hyperCKemia in a case of Danon disease due to a missense mutation in Lamp-2 gene. Musumeci O; Rodolico C; Nishino I; Di Guardo G; Migliorato A; Aguennouz M; Mazzeo A; Messina C; Vita G; Toscano A Neuromuscul Disord; 2005 Jun; 15(6):409-11. PubMed ID: 15907287 [TBL] [Abstract][Full Text] [Related]
15. Is myopathy with rimmed vacuoles a hallmark of juvenile neuronal ceroid lipofuscinosis (CLN3)? Borgione E; Castello F; Lo Giudice M; Santa Paola S; Salvatore S; Berti G; Malandrini A; Bottitta M; Musumeci SA; Scuderi C Neurol Sci; 2016 May; 37(5):805-7. PubMed ID: 26700800 [No Abstract] [Full Text] [Related]
17. Autophagy is disrupted in a knock-in mouse model of juvenile neuronal ceroid lipofuscinosis. Cao Y; Espinola JA; Fossale E; Massey AC; Cuervo AM; MacDonald ME; Cotman SL J Biol Chem; 2006 Jul; 281(29):20483-93. PubMed ID: 16714284 [TBL] [Abstract][Full Text] [Related]
18. [Eludication of pathomechanism of and development of therapy for autophagic vacuolar myopathies]. Nishino I Rinsho Shinkeigaku; 2010 Jan; 50(1):1-6. PubMed ID: 20120346 [TBL] [Abstract][Full Text] [Related]
19. Novel homozygous CLN3 missense variant in isolated retinal dystrophy: A case report and electron microscopic findings. Mizobuchi K; Hayashi T; Yoshitake K; Fujinami K; Tachibana T; Tsunoda K; Iwata T; Nakano T Mol Genet Genomic Med; 2020 Aug; 8(8):e1308. PubMed ID: 32441891 [TBL] [Abstract][Full Text] [Related]
20. Characterizing upper limb function in the context of activities of daily living in CLN3 disease. Hildenbrand H; Wickstrom J; Parks R; Zampieri C; Nguyen TT; Thurm A; Jenkins K; Alter KE; Matsubara J; Hammond D; Soldatos A; Porter FD; Dang Do AN Am J Med Genet A; 2021 May; 185(5):1399-1413. PubMed ID: 33559393 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]