Latest NICE draft guidance does not recommend elusulfase alfa for rare disorder

2021 ◽  
Vol 892 (1) ◽  
pp. 32-32
Keyword(s):  
2010 ◽  
Vol 41 (6) ◽  
pp. 9
Author(s):  
JANE ANDERSON
Keyword(s):  

2012 ◽  
Vol 43 (3) ◽  
pp. 6 ◽  
Author(s):  
ALICIA AULT
Keyword(s):  

Blood ◽  
2017 ◽  
Vol 130 (12) ◽  
pp. 1402-1408 ◽  
Author(s):  
Guy Young

Abstract The incidence of pediatric venous thromboembolism (VTE) has been increasing significantly over the past decade in part as a result of increased recognition of this serious disorder but more so because of the increased use of central venous catheters and other technological advancements involved in the care of ill children. Management of pediatric VTE is a complex undertaking, considering that the vast majority of children who develop this complication have serious underlying medical disorders. Although the incidence is rising, in comparison with adults, this remains a relatively rare disorder, and as such, large-scale clinical trials have not been completed, rendering management decisions to be based on extrapolation from adult data and the experience of the treating physician. Clearly, both are fraught with problems. Thus, day-to-day management remains more art than science until such time that the results from clinical trials (many of which are under way) become available. This edition of “How I Treat” describes the author’s experience in managing 3 common scenarios that one may encounter in pediatric thrombosis and suggests a logical approach to such situations. Furthermore, the author provides 3 algorithms to help guide management decisions.


2012 ◽  
Vol 36 (3) ◽  
pp. 297-300 ◽  
Author(s):  
K Sawaki ◽  
K Mishima ◽  
A Sato ◽  
Y Goda ◽  
A Osugi ◽  
...  

Zimmermann–Laband syndrome is a very rare disorder characterized by gingival fibromatosis, abnormalities of soft cartilages of the nose and/or ears, hypoplastic or absent nails and terminal phalanges, joint hypermobility, hypatoslenomegaly, mild hirsutism and learning difficulties. Early presentation of Zimmer–mann–Laband syndrome in a newborn has rarely been described. This paper describes a newborn patient with Zimmermann–Laband syndrome.


2017 ◽  
Vol 9 (2) ◽  
pp. 91-97
Author(s):  
Sara Ghoneim ◽  
Martin Zaiac

A 37-year-old South-Asian male presented to our clinic with a crusty, verrucous-like, scaly plaque of the left ala of the nose. After ruling out infectious and other epidermal bullous diseases, we finalized a diagnosis of localized pemphigus foliaceus, an exceptionally rare disorder with only 15 cases reported in the literature to date. The hyperkeratotic lesions responded favorably to a 3-week regimen of triamcinolone ointment and a onetime intralesional triamcinolone 2.5 mg/mL injection.


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