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Weston P Miller

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Articles 33
Citations 795
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Recent Articles
1.
Nguyen A, Miller W, Gupta A, Lund T, Schiferl D, Lam L, et al.
JBMR Plus . 2022 Mar; 6(3):e10597. PMID: 35309862
The only treatment currently available for patients with severe infantile osteopetrosis is hematopoietic cell transplantation (HCT). HCT-related toxicity and mortality risks typically preclude its use in non-infantile patients, and other...
2.
Galvin R, Cao Q, Miller W, Knight-Perry J, Smith A, Ebens C
Transplant Cell Ther . 2021 Apr; 27(4):316.e1-316.e8. PMID: 33836874
Immune-mediated cytopenias (IMC)-isolated or combined hemolytic anemia, thrombocytopenia, or neutropenia-are increasingly recognized as serious complications after allogeneic hematopoietic cell transplantation (HCT) for nonmalignant disorders (NMD). However, IMC incidence, duration, response...
3.
Lum S, Orchard P, Lund T, Miller W, Boelens J, Wynn R
Transplant Cell Ther . 2020 Sep; 27(1):91.e1-91.e4. PMID: 32961374
We report the outcomes of cord blood transplantation (CBT) with a busulfan (Bu) pharmacokinetics-targeted myeloablative conditioning regimen in 97 children with Hurler syndrome (HS) performed between 2004 and 2016. The...
4.
Pierpont E, Nascene D, Shanley R, Kenney-Jung D, Ziegler R, Miller W, et al.
Neurology . 2020 Jul; 95(5):e591-e600. PMID: 32616675
Objective: To quantify benchmark treatment outcomes that may be enabled by newborn screening surveillance for X-linked adrenoleukodystrophy (ALD), we report neurocognitive, neuropsychiatric, and MRI change for boys who underwent hematopoietic...
5.
Gupta A, Orchard P, Miller W, Nascene D, Raymond G, Loes D, et al.
Stem Cells Transl Med . 2020 Feb; 9(5):554-558. PMID: 32020747
Cerebral adrenoleukodystrophy is an inflammatory demyelinating condition that is the result of a mutation in the X-linked ABCD1 gene, a peroxisomal very long chain fatty acid transporter. Although mutations in...
6.
Gupta A, Downey M, Shanley R, Jennissen C, Miller W, Lund T, et al.
Biol Blood Marrow Transplant . 2019 Nov; 26(3):486-492. PMID: 31751770
Hematopoietic stem cell transplantation (HCT) is a primary treatment for various inherited metabolic disorders (IMDs). Achieving stable and sustained engraftment while minimizing transplantation-related morbidity and mortality is critical to optimizing...
7.
Lund T, Miller W, Liao A, Tolar J, Shanley R, Pasquali M, et al.
Sci Rep . 2019 Oct; 9(1):14105. PMID: 31575939
Allogeneic hematopoietic cell transplantation (HCT) benefits children with Hurler syndrome (MPS-IH). However, survivors remain burdened by substantial MPS-IH related residual disease. We studied the feasibility, safety and biochemical impact of...
8.
Polgreen L, Lund T, Braunlin E, Tolar J, Miller B, Fung E, et al.
Pediatr Res . 2019 Aug; 87(1):104-111. PMID: 31434105
Background: Mucopolysaccharidosis I (MPS IH) is a lysosomal storage disease treated with hematopoietic cell transplantation (HCT) because it stabilizes cognitive deterioration, but is insufficient to alleviate all somatic manifestations. Intravenous...
9.
Orchard P, Markowski T, Higgins L, Raymond G, Nascene D, Miller W, et al.
Sci Rep . 2019 May; 9(1):7858. PMID: 31133696
Cerebral adrenoleukodystrophy (cALD) is an inflammatory neurodegenerative disease associated with mutation of the ABCD1 gene. Proteomic analysis of cerebral spinal fluid (CSF) from young males with active cALD revealed markers...
10.
Lund T, Miller W, Eisengart J, Simmons K, Pollard L, Renaud D, et al.
Mol Genet Genomic Med . 2019 May; 7(7):e00712. PMID: 31115173
Background: Deficiency in the enzyme β-mannosidase was described over three decades ago. Although rare in occurrence, the presentation of childhood-onset β-mannosidase deficiency consists of hypotonia in the newborn period followed...