Literature DB >> 15990985

[Adult Refsum disease. A retinal dystrophy with therapeutic options].

K Rüther1.   

Abstract

Adult Refsum disease is one of the few forms of tapetoretinal degenerations accessible for therapy. The disease is characterized by an elevated plasma phytanic acid level and high concentrations of phytanic acid in a variety tissues. Beside tapetoretinal degeneration, additional symptoms are chronic polyneuropathy, cerebellar ataxia, sensorineural hearing loss, anosmia, ichthyosis, skeletal malformations, and cardiac abnormalities. A diet low in phytanic acid leads to an amelioration of polyneuropathy and ataxia and slows or even stops the other manifestations. This beneficial effect of dietary precautions requires the need to get hold of as much patients as possible but better all of them. The ophthalmologist plays a crucial role to this end because of the early manifestation of the tapetoretinal degeneration. A delay of 11 years between the appearance of first symptoms and the diagnosis of Refsum disease, as reported in the literature, is not acceptable.

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Year:  2005        PMID: 15990985     DOI: 10.1007/s00347-005-1239-x

Source DB:  PubMed          Journal:  Ophthalmologe        ISSN: 0941-293X            Impact factor:   1.059


  20 in total

1.  Phytanoyl-coenzyme A hydroxylase deficiency -- the enzyme defect in Refsum's disease.

Authors:  G A Jansen; R J Wanders; P A Watkins; S J Mihalik
Journal:  N Engl J Med       Date:  1997-07-10       Impact factor: 91.245

2.  Identification of PAHX, a Refsum disease gene.

Authors:  S J Mihalik; J C Morrell; D Kim; K A Sacksteder; P A Watkins; S J Gould
Journal:  Nat Genet       Date:  1997-10       Impact factor: 38.330

3.  Effects of phytanic acid on the vitamin E status, lipid composition and physical properties of retinal cell membranes: implications for adult Refsum disease.

Authors:  S P Young; A W Johnson; D P Muller
Journal:  Clin Sci (Lond)       Date:  2001-12       Impact factor: 6.124

4.  [Course of Refsum's disease under diabetic treatment. Clinical, biochemical and neuropathological data (author's transl)].

Authors:  H Lenz; E Sluga; H Bernheimer; B Molzer; W Pürgyi
Journal:  Nervenarzt       Date:  1979-01       Impact factor: 1.214

5.  Dietary effects on serum-phytanic-acid levels and on clinical manifestations in heredopathia atactica polyneuritiformis.

Authors:  L Eldjarn; K Try; O Stokke; A W Munthe-Kaas; S Refsum; D Steinberg; J Avigan; C Mize
Journal:  Lancet       Date:  1966-03-26       Impact factor: 79.321

6.  Omega-hydroxylation of phytanic acid in rat liver microsomes: implications for Refsum disease.

Authors:  J C Komen; M Duran; R J A Wanders
Journal:  J Lipid Res       Date:  2004-04-21       Impact factor: 5.922

7.  Phytol metabolites are circulating dietary factors that activate the nuclear receptor RXR.

Authors:  S Kitareewan; L T Burka; K B Tomer; C E Parker; L J Deterding; R D Stevens; B M Forman; D E Mais; R A Heyman; T McMorris; C Weinberger
Journal:  Mol Biol Cell       Date:  1996-08       Impact factor: 4.138

8.  Heredopathia atactica polyneuritiformis (Refsum's disease). Experiences of dietary treatment and plasmapheresis.

Authors:  A Lundberg; L G Lilja; P O Lundberg; K Try
Journal:  Eur Neurol       Date:  1972       Impact factor: 1.710

9.  Heredopathia atactica polyneuritiformis (Refsum's disease): 2. Estimation of phytanic acid in foods.

Authors:  A Masters-Thomas; J Bailes; J D Billimoria; M E Clemens; F B Gibberd; N G Page
Journal:  J Hum Nutr       Date:  1980-08

10.  Heredopathia atactica polyneuritiformis (Refsum's disease): 1. Clinical features and dietary management.

Authors:  A Masters-Thomas; J Bailes; J D Billimoria; M E Clemens; F B Gibberd; N G Page
Journal:  J Hum Nutr       Date:  1980-08
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  2 in total

1.  Refsum's Disease-Use of the Intestinal Lipase Inhibitor, Orlistat, as a Novel Therapeutic Approach to a Complex Disorder.

Authors:  Nimalie J Perera; Barry Lewis; Huy Tran; Michael Fietz; David R Sullivan
Journal:  J Obes       Date:  2010-09-01

2.  The Challenges of a Successful Pregnancy in a Patient with Adult Refsum's Disease due to Phytanoyl-CoA Hydroxylase Deficiency.

Authors:  Karolina M Stepien; Anthony S Wierzbicki; Bwee T Poll-The; Hans R Waterham; Christian J Hendriksz
Journal:  JIMD Rep       Date:  2016-08-13
  2 in total

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