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Items: 1 to 20 of 111

1.

Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease.

Tolar J, Petryk A, Khan K, Bjoraker KJ, Jessurun J, Dolan M, Kivisto T, Charnas L, Shapiro EG, Orchard PJ.

Bone Marrow Transplant. 2009 Jan;43(1):21-7. doi: 10.1038/bmt.2008.273. Epub 2008 Sep 8.

PMID:
18776925
2.

Wolman disease successfully treated by bone marrow transplantation.

Krivit W, Peters C, Dusenbery K, Ben-Yoseph Y, Ramsay NK, Wagner JE, Anderson R.

Bone Marrow Transplant. 2000 Sep;26(5):567-70.

3.

[Wolman disease].

Mnif K, Chabchoub A, Marrakchi Z, Ayachi R, Khrouf N.

Arch Pediatr. 1994 Jul;1(7):663-6. French.

PMID:
7987466
4.

Pathological evidence of Wolman's disease following hematopoietic stem cell transplantation despite correction of lysosomal acid lipase activity.

Gramatges MM, Dvorak CC, Regula DP, Enns GM, Weinberg K, Agarwal R.

Bone Marrow Transplant. 2009 Oct;44(7):449-50. doi: 10.1038/bmt.2009.57. Epub 2009 Mar 23. No abstract available.

PMID:
19308038
5.

A case of acid lipase deficiency: Wolman's disease.

Bona G, Bracco G, Gallina MR, Iavarone A, Artesani L, Perona A, Zaffaroni M.

Panminerva Med. 1989 Jan-Mar;31(1):49-53.

PMID:
2726290
6.

Unfavorable outcome of hematopoietic stem cell transplantation in two siblings with Wolman disease due to graft failure and hepatic complications.

Yanir A, Allatif MA, Weintraub M, Stepensky P.

Mol Genet Metab. 2013 Jun;109(2):224-6. doi: 10.1016/j.ymgme.2013.03.007. Epub 2013 Mar 20.

PMID:
23583223
7.

Isolated fetal ascites caused by Wolman disease.

Ben-Haroush A, Yogev Y, Levit O, Hod M, Kaplan B.

Ultrasound Obstet Gynecol. 2003 Mar;21(3):297-8.

8.

Relative survival of long-term hematopoietic cell transplant recipients approaches general population rates.

Nivison-Smith I, Simpson JM, Dodds AJ, Ma DD, Szer J, Bradstock KF.

Biol Blood Marrow Transplant. 2009 Oct;15(10):1323-30. doi: 10.1016/j.bbmt.2009.06.014. Epub 2009 Aug 3.

9.

[Wolman disease with novel mutation of LIPA gene in a Chinese infant].

Huang YL, Sheng HY, Zhao XY, Yu JK, Li L, Liu HS, Gu CM, He DM, Liu L.

Zhonghua Er Ke Za Zhi. 2012 Aug;50(8):601-5. Chinese.

PMID:
23158738
10.

Patients with early relapse of primary hemophagocytic syndromes or with persistent CNS involvement may benefit from immediate hematopoietic stem cell transplantation.

Sparber-Sauer M, Hönig M, Schulz AS, zur Stadt U, Schütz C, Debatin KM, Friedrich W.

Bone Marrow Transplant. 2009 Sep;44(6):333-8. doi: 10.1038/bmt.2009.34. Epub 2009 Mar 2.

PMID:
19252534
11.

Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants.

Jones SA, Valayannopoulos V, Schneider E, Eckert S, Banikazemi M, Bialer M, Cederbaum S, Chan A, Dhawan A, Di Rocco M, Domm J, Enns GM, Finegold D, Gargus JJ, Guardamagna O, Hendriksz C, Mahmoud IG, Raiman J, Selim LA, Whitley CB, Zaki O, Quinn AG.

Genet Med. 2016 May;18(5):452-8. doi: 10.1038/gim.2015.108. Epub 2015 Aug 27.

12.

Hypertension and diabetes mellitus in adult and pediatric survivors of allogeneic hematopoietic cell transplantation.

Majhail NS, Challa TR, Mulrooney DA, Baker KS, Burns LJ.

Biol Blood Marrow Transplant. 2009 Sep;15(9):1100-7. doi: 10.1016/j.bbmt.2009.05.010.

13.

[Diagnostic image (358). A neonate with splenomegaly and calcified adrenal glands].

Pot DJ, Kardux JJ.

Ned Tijdschr Geneeskd. 2008 Jan 26;152(4):202. Dutch.

PMID:
18320945
14.

Wolman disease: morphological, clinical and genetic studies on the first Scandinavian cases.

Röyttä M, Fagerlund AS, Toikkanen S, Salmi TT, Jorde LB, Forsius HR, Eriksson AW.

Clin Genet. 1992 Jul;42(1):1-7.

PMID:
1516222
15.

[Wolman's disease in an infant].

Storm W, Wendel U, Sprenkamp M, Seidler A.

Monatsschr Kinderheilkd. 1990 Feb;138(2):88-90. German.

PMID:
2320017
16.

Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.

Jones SA, Rojas-Caro S, Quinn AG, Friedman M, Marulkar S, Ezgu F, Zaki O, Gargus JJ, Hughes J, Plantaz D, Vara R, Eckert S, Arnoux JB, Brassier A, Le Quan Sang KH, Valayannopoulos V.

Orphanet J Rare Dis. 2017 Feb 8;12(1):25. doi: 10.1186/s13023-017-0587-3.

17.

Outcomes of 5-year survivors of pediatric liver transplantation: report on 461 children from a north american multicenter registry.

Ng VL, Fecteau A, Shepherd R, Magee J, Bucuvalas J, Alonso E, McDiarmid S, Cohen G, Anand R; Studies of Pediatric Liver Transplantation Research Group.

Pediatrics. 2008 Dec;122(6):e1128-35. doi: 10.1542/peds.2008-1363.

PMID:
19047213
18.

Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation.

Stein J, Garty BZ, Dror Y, Fenig E, Zeigler M, Yaniv I.

Eur J Pediatr. 2007 Jul;166(7):663-6. Epub 2006 Oct 11.

PMID:
17033804
19.

Infant case of lysosomal acid lipase deficiency: Wolman's disease.

Sadhukhan M, Saha A, Vara R, Bhaduri B.

BMJ Case Rep. 2014 May 15;2014. pii: bcr2013202652. doi: 10.1136/bcr-2013-202652.

20.

Long-term outcome of allogeneic stem cell transplantation in chronic lymphocytic leukemia: analysis after a minimum follow-up of 5 years.

Malhotra P, Hogan WJ, Litzow MR, Elliott MA, Gastineau DA, Ansell SM, Dispenzieri A, Gertz MA, Hayman SR, Inwards DJ, Lacy MQ, Micallef IN, Porrata LF, Tefferi A.

Leuk Lymphoma. 2008 Sep;49(9):1724-30. doi: 10.1080/10428190802263535.

PMID:
18798106

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