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1 . Academic Journal
Major clinical events and healthcare resource use among patients with long-chain fatty acid oxidation disorders in the United States: Results from LC-FAOD Odyssey program
저자
by
Yang, Erru
;
Kruger, Eliza
;
Yin, David
;
Mace, Kieran
;
Tierney, Meghan
, et al.
소스
In
Molecular Genetics and Metabolism
May 2024 142(1)
Full Text (ScienceDirect)
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7 . Academic Journal
Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?
저자
by
Grant, Christina L.
;
López-Valdez, Jaime
;
Marsden, Deborah
;
Ezgü, Fatih
.
소스
In
Molecular Genetics and Metabolism
March 2024 141(3)
Full Text (ScienceDirect)
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6 . Academic Journal
Growth patterns in patients with mucopolysaccharidosis VII
저자
by
Montaño, Adriana M.
;
Różdżyńska-Świątkowska, Agnieszka
;
Jurecka, Agnieszka
;
Ramirez, Antonio Nino
;
Zhang, Lin
, et al.
소스
In
Molecular Genetics and Metabolism Reports
September 2023 36
Full Text (ScienceDirect)
Full Text (ScienceDirect O/A)
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7 . Academic Journal
Clinical manifestations, healthcare resource utilization, and costs among patients with long-chain fatty acid oxidation disorders: a retrospective claims database analysis.
저자
by
Yang, Erru (Christy)
;
Fang, Zhenzhen
;
Tan, Ruixin
;
Guo, Yun
;
He, Siyi
, et al.
소스
Current Medical Research & Opinion
. Sep2024, p1-14. 14p. 3 Illustrations, 7 Charts.
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7 . Academic Journal
Parallel biochemical and genetic testing informs a timely and accurate diagnosis of MPS VII: Findings from 5 years of sponsored testing programs
저자
by
Miller, Vanessa Rangel
;
Daugherty, Sean
;
Gupta, Rupal N.
;
Hegde, Madhuri
;
Johnson, Jennifer
, et al.
소스
In
Molecular Genetics and Metabolism
February 2024 141(2)
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7 . Academic Journal
Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)
저자
by
Malkus, Betsy C.
;
Cohen, Jeanne M.
;
Grant, Christina L.
;
Scarpa, Maurizio
;
Sun, Angela
, et al.
소스
In
Molecular Genetics and Metabolism
February 2024 141(2)
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7 . Academic Journal
Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multi-center disease monitoring program (DMP)
저자
by
Giugliani, Roberto
;
Grant, Christina L.
;
Scarpa, Maurizio
;
Sun, Angela
;
Wang, Raymond Y.
, et al.
소스
In
Molecular Genetics and Metabolism
February 2024 141(2)
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7 . Academic Journal
P261: Vestronidase alfa for the treatment of mucopolysaccharidosis VII (MPS VII): Updated results from a longitudinal, multicenter disease monitoring program (DMP)
저자
by
Giugliani, Roberto
;
Gonzalez-Meneses, Antonio
;
Grant, Christina
;
Scarpa, Maurizio
;
Sun, Angela
, et al.
소스
In
Genetics in Medicine Open
2024 2 Supplement 1
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7 . Academic Journal
P030: Diverse genetic findings, clinical signs, and newborn screening history from a long-chain fatty acid oxidation disorders gene panel sponsored program
저자
by
Miller, Nicole
;
Gutierrez, Heraclio
;
Japalaghi, Omid
;
Konczal, Laura
;
Marsden, Deborah
, et al.
소스
In
Genetics in Medicine Open
2024 2 Supplement 1
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7 . Academic Journal
P032: Decoding the genetic tapestry of long chain fatty acid oxidation disorders: Unveiling novel insights with a dynamic locus-specific gene database
저자
by
Miller, Vanessa Rangel
;
Richbourg, Heather
;
Vockley, Jerry
;
Japalaghi, Omid
;
AlSayed, Moeenaldeen
, et al.
소스
In
Genetics in Medicine Open
2024 2 Supplement 1
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