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Page 1
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
N Engl J Med. 2024 Feb 1;390(5):421-431. doi: 10.1056/NEJMoa2310151.
N Engl J Med. 2024.
PMID: 38294974
Clinical Trial.
Quantitative longitudinal natural history of 8 gangliosidoses-conceptual framework and baseline data of the German 8-in-1 disease registry. A cross-sectional analysis.
Ries M, Mendoza G, Arash-Kaps L, Amraoui Y, Quack F, Hardt B, Diederich S, Beck M, Mengel E.
Ries M, et al. Among authors: arash kaps l.
Genet Med. 2022 Dec;24(12):2434-2443. doi: 10.1016/j.gim.2022.09.001. Epub 2022 Oct 4.
Genet Med. 2022.
PMID: 36194207
Free article.
Item in Clipboard
A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.
Wasserstein M, Lachmann R, Hollak C, Arash-Kaps L, Barbato A, Gallagher RC, Giugliani R, Guelbert NB, Ikezoe T, Lidove O, Mabe P, Mengel E, Scarpa M, Senates E, Tchan M, Villarrubia J, Chen Y, Furey S, Thurberg BL, Zaher A, Kumar M.
Wasserstein M, et al. Among authors: arash kaps l.
Genet Med. 2022 Jul;24(7):1425-1436. doi: 10.1016/j.gim.2022.03.021. Epub 2022 Apr 26.
Genet Med. 2022.
PMID: 35471153
Free article.
Clinical Trial.
Item in Clipboard
The Clinical and Molecular Spectrum of GM1 Gangliosidosis.
Arash-Kaps L, Komlosi K, Seegräber M, Diederich S, Paschke E, Amraoui Y, Beblo S, Dieckmann A, Smitka M, Hennermann JB.
Arash-Kaps L, et al.
J Pediatr. 2019 Dec;215:152-157.e3. doi: 10.1016/j.jpeds.2019.08.016.
J Pediatr. 2019.
PMID: 31761138
Item in Clipboard
Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III).
Muschol NM, Pape D, Kossow K, Ullrich K, Arash-Kaps L, Hennermann JB, Stücker R, Breyer SR.
Muschol NM, et al. Among authors: arash kaps l.
Orphanet J Rare Dis. 2019 May 2;14(1):93. doi: 10.1186/s13023-019-1065-x.
Orphanet J Rare Dis. 2019.
PMID: 31046785
Free PMC article.
Item in Clipboard
Pharmacokinetics, pharmacodynamics, and safety of moss-aGalactosidase A in patients with Fabry disease.
Hennermann JB, Arash-Kaps L, Fekete G, Schaaf A, Busch A, Frischmuth T.
Hennermann JB, et al. Among authors: arash kaps l.
J Inherit Metab Dis. 2019 May;42(3):527-533. doi: 10.1002/jimd.12052. Epub 2019 Feb 11.
J Inherit Metab Dis. 2019.
PMID: 30746723
Clinical Trial.
Item in Clipboard
Retinal and optic nerve degeneration in α-mannosidosis.
Matlach J, Zindel T, Amraoui Y, Arash-Kaps L, Hennermann JB, Pitz S.
Matlach J, et al. Among authors: arash kaps l.
Orphanet J Rare Dis. 2018 Jun 1;13(1):88. doi: 10.1186/s13023-018-0829-z.
Orphanet J Rare Dis. 2018.
PMID: 29859105
Free PMC article.
Item in Clipboard
Impact of immunosuppressive therapy on therapy-neutralizing antibodies in transplanted patients with Fabry disease.
Lenders M, Oder D, Nowak A, Canaan-Kühl S, Arash-Kaps L, Drechsler C, Schmitz B, Nordbeck P, Hennermann JB, Kampmann C, Reuter S, Brand SM, Wanner C, Brand E.
Lenders M, et al. Among authors: arash kaps l.
J Intern Med. 2017 Sep;282(3):241-253. doi: 10.1111/joim.12647. Epub 2017 Jul 26.
J Intern Med. 2017.
PMID: 28682471
Free article.
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