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New spastic paraplegia phenotype associated to mutation of NFU1.
Tonduti, Davide; Dorboz, Imen; Imbard, Apolline; Slama, Abdelhamid; Boutron, Audrey; Pichard, Samia; Elmaleh, Monique; Vallée, Louis; Benoist, Jean François; Ogier, Heléne; Boespflug-Tanguy, Odile.
Afiliação
  • Tonduti D; Paris Diderot University - Sorbonne Paris Cité; Inserm U1141, DHU PROTECT, Robert Debré Hospital, Paris, France. davidetondu@hotmail.com.
  • Dorboz I; Department of Brain and Behavioral Sciences, Unit of Child Neurology and Psychiatry, University of Pavia, Pavia, Italy. davidetondu@hotmail.com.
  • Imbard A; Paris Diderot University - Sorbonne Paris Cité; Inserm U1141, DHU PROTECT, Robert Debré Hospital, Paris, France. imen.dorboz@inserm.fr.
  • Slama A; Hormonology and Biochemistry Departement, Robert Debré Hospital, AP-HP, Paris, France. apolline.imbard@rdb.aphp.fr.
  • Boutron A; Department of Biochemistry, Bicetre Hospital, AP-HP, Le Kremlin Bicetre, France. abdel.slama@bct.aphp.fr.
  • Pichard S; Department of Biochemistry, Bicetre Hospital, AP-HP, Le Kremlin Bicetre, France. audrey.boutron@bct.aphp.fr.
  • Elmaleh M; Departement of Neuropediatrics and Metabolic Diseases, Robert Debré Hospital, AP-HP, 48, Boulevard Sérurier, 75019, Paris, France. samia.pichard@rdb.aphp.fr.
  • Vallée L; Departement of Pediatric Radiology, Robert Debré Hospital, AP-HP, Paris, France. monique.elmaleh@rdb.aphp.fr.
  • Benoist JF; Department of Neuropediatrics, CHRU, University Lille North, Lille, France. louis.vallee@chru-lille.fr.
  • Ogier H; Hormonology and Biochemistry Departement, Robert Debré Hospital, AP-HP, Paris, France. jean-francois.benoist@rdb.aphp.fr.
  • Boespflug-Tanguy O; Departement of Neuropediatrics and Metabolic Diseases, Robert Debré Hospital, AP-HP, 48, Boulevard Sérurier, 75019, Paris, France. helene.ogier@rdb.aphp.fr.
Orphanet J Rare Dis ; 10: 13, 2015 Feb 08.
Article em En | MEDLINE | ID: mdl-25758857
Recently an early onset lethal encephalopathy has been described in relation to mutations of NFU1, one of the genes involved in iron-sulfur cluster metabolism. We report a new NFU1 mutated patient presenting with a milder phenotype characterized by a later onset, a slowly progressive spastic paraparesis with relapsing-remitting episodes, mild cognitive impairment and a long survival. The early white matter abnormalities observed on MRI was combined with a mixed sensory-motor neuropathy in the third decade. Our case clearly suggests the importance of considering NFU1 mutation in slowly evolving leukoencephalopathy with high glycine concentration.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas de Transporte / Paraparesia Espástica Tipo de estudo: Risk_factors_studies Limite: Adult / Humans / Male Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2015 Tipo de documento: Article País de afiliação: França País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas de Transporte / Paraparesia Espástica Tipo de estudo: Risk_factors_studies Limite: Adult / Humans / Male Idioma: En Revista: Orphanet J Rare Dis Assunto da revista: MEDICINA Ano de publicação: 2015 Tipo de documento: Article País de afiliação: França País de publicação: Reino Unido