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A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.
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.
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
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
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.
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
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
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
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
Bremova-Ertl T, Ramaswami U, Brands M, Foltan T, Gautschi M, Gissen P, Gowing F, Hahn A, Jones S, Kay R, Kolnikova M, Arash-Kaps L, Marquardt T, Mengel E, Park JH, Reichmannová S, Schneider SA, Sivananthan S, Walterfang M, Wibawa P, Strupp M, Martakis K.
Bremova-Ertl T, et al. Among authors: arash kaps l.
N Engl J Med. 2024 Feb 1;390(5):421-431. doi: 10.1056/NEJMoa2310151.
N Engl J Med. 2024.
PMID: 38294974
Clinical Trial.
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