Metreleptin treatment for congenital generalized lipodystrophy type 4 (CGL4): a case report.

PTRF congenital generalized lipodystrophy leptin metreleptin

Journal

Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology
ISSN: 0918-5739
Titre abrégé: Clin Pediatr Endocrinol
Pays: Japan
ID NLM: 9433330

Informations de publication

Date de publication:
2019
Historique:
received: 09 05 2018
accepted: 31 08 2018
entrez: 13 2 2019
pubmed: 13 2 2019
medline: 13 2 2019
Statut: ppublish

Résumé

Congenital generalized lipodystrophy type 4 (CGL4) is a rare disease caused by mutations in the gene polymerase I and transcript release factor (

Identifiants

pubmed: 30745727
doi: 10.1297/cpe.28.1
pii: 2018-0015
pmc: PMC6356095
doi:

Types de publication

Journal Article

Langues

eng

Pagination

1-7

Références

Gut. 1975 Mar;16(3):230-4
pubmed: 1123178
N Engl J Med. 2002 Feb 21;346(8):570-8
pubmed: 11856796
J Clin Endocrinol Metab. 1954 Feb;14(2):193-204
pubmed: 13130666
J Clin Endocrinol Metab. 2007 Feb;92(2):532-41
pubmed: 17118991
J Clin Invest. 2009 Sep;119(9):2623-33
pubmed: 19726876
PLoS Genet. 2010 Mar 12;6(3):e1000874
pubmed: 20300641
Mol Genet Metab. 2010 Oct-Nov;101(2-3):233-7
pubmed: 20638880
Am J Med Genet A. 2010 Sep;152A(9):2245-53
pubmed: 20684003
J Clin Endocrinol Metab. 2011 Nov;96(11):3313-25
pubmed: 21865368
Neuromuscul Disord. 2013 May;23(5):441-4
pubmed: 23489663
BMC Med Genet. 2013 Sep 11;14:89
pubmed: 24024685
Metabolism. 2015 Jan;64(1):24-34
pubmed: 25199978
Int J Obes (Lond). 2014 Sep;38 Suppl 2:S32-8
pubmed: 25219410
Eur J Med Genet. 2015 Apr;58(4):216-21
pubmed: 25721873
Expert Opin Biol Ther. 2015 Jul;15(7):1061-75
pubmed: 26063386
Ther Clin Risk Manag. 2015 Sep 16;11:1391-400
pubmed: 26396524
Clin Endocrinol (Oxf). 2016 Jul;85(1):137-49
pubmed: 26589105
Ir J Med Sci. 1981 Mar;150(3):84-5
pubmed: 7228604

Auteurs

Shinji Takeyari (S)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Satoshi Takakuwa (S)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
Department of Pediatrics, Hyogo Prefectural Nishinomiya Hospital, Hyogo, Japan.

Kei Miyata (K)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Kenichi Yamamoto (K)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
Department of Statistical Genetics, Osaka University Graduate School of Medicine, Osaka, Japan.

Hirofumi Nakayama (H)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
The Japan Environment and Children's Study, Osaka Unit Center, Osaka, Japan.

Yasuhisa Ohata (Y)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
The 1 Department of Oral and Maxillofacial Surgery, Osaka University Graduate School of Dentistry, Osaka, Japan.

Makoto Fujiwara (M)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Taichi Kitaoka (T)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Takuo Kubota (T)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Noriyuki Namba (N)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization (JCHO), Osaka, Japan.

Norio Sakai (N)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.
Child Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, Osaka, Japan.

Keiichi Ozono (K)

Department of Pediatrics, Osaka University Graduate School of Medicine, Osaka, Japan.

Classifications MeSH