| Literature DB >> 30569574 |
Ken Saida1, Sebastian Silva2, Benjamin Solar2, Atsushi Fujita1, Kohei Hamanaka1, Satomi Mitsuhashi1, Eriko Koshimizu1, Takeshi Mizuguchi1, Satoko Miyatake1, Atsushi Takata1, Noriko Miyake1, Naomichi Matsumoto1.
Abstract
SOFT syndrome (MIM614813) is an extremely rare primordial dwarfism caused by biallelic mutations in the POC1A gene. It is characterized by prenatal short stature, onychodysplasia, facial dysmorphism, hypotrichosis, and variable skeletal abnormalities including hypoplastic pelvis and sacrum, small hands, and cone-shaped epiphyses, as well as delayed bone age. To the best of our knowledge, only eight POC1A mutations have been reported in humans to date. We report a 7-year-old Chilean girl with SOFT syndrome arising from a novel POC1A mutation c. 649C>T, p.Arg217Trp. Although her clinical features were largely compatible with SOFT syndrome, hand X-ray examinations at 3.5 and 6 years unexpectedly showed normal bone age. Automated bone age determination was performed using image analysis software, BoneXpert. This case highlights the importance of the accumulation of patients with POC1A mutations to further elucidate the detailed clinical features of SOFT syndrome.Entities:
Keywords: zzm321990POC1A; SOFT syndrome; bone age; patent ductus arteriosus
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Year: 2018 PMID: 30569574 DOI: 10.1002/ajmg.a.61015
Source DB: PubMed Journal: Am J Med Genet A ISSN: 1552-4825 Impact factor: 2.802