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JIMD Reports
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November 18, 2020
Fabry disease in India: A multicenter study of the clinical and mutation spectrum in 54 patients
Sheela Nampoothiri, Dhanya Yesodharan, Amrita Bhattacherjee, et al.
JIMD Reports
|
November 18, 2020
Cerebrotendinous xanthomatosis-associated diarrhea and response to chenodeoxycholic acid treatment
Eric P Brass, Bianca M L Stelten, Aad Verrips
JIMD Reports
|
February 20, 2020
Early detection of lysosomal diseases by screening of cases of idiopathic splenomegaly and/or thrombocytopenia with a next-generation sequencing gene panel
Gloria Muñoz, David García-Seisdedos, Crina Ciubotariu, et al.
JIMD Reports
|
February 20, 2020
NGLY1 deficiency: Novel patient, review of the literature and diagnostic algorithm
Patryk Lipiński, Anna Bogdańska, Agnieszka Różdżyńska-Świątkowska, et al.
JIMD Reports
|
February 20, 2020
Neurotoxicity including posterior reversible encephalopathy syndrome after initiation of calcineurin inhibitors in transplanted methylmalonic acidemia patients: Two case reports and review of the literature
Femke Molema, Monique Williams, Janneke Langendonk, et al.
JIMD Reports
|
March 11, 2018
Cognitive and Behavioural Outcomes of Paediatric Liver Transplantation for Ornithine Transcarbamylase Deficiency
Louise Crowe, Vicki Anderson, Winita Hardikar, et al.
JIMD Reports
|
January 31, 2018
Beneficial Effect of BH<sub>4</sub> Treatment in a 15-Year-Old Boy with Biallelic Mutations in DNAJC12
Monique G M de Sain-van der Velden, Willemijn F E Kuper, Marie-Anne Kuijper, et al.
JIMD Reports
|
December 16, 2017
Expert Opinion vs Patient Perspective in Treatment of Rare Disorders: Tooth Removal in Lesch-Nyhan Disease as an Example
Adam C Cotton, R B Bell, H A Jinnah
JIMD Reports
|
March 11, 2020
Cavitating and tigroid-like leukoencephalopathy in a case of <i>NDUFA2</i>-related disorder
Marianna Alagia, Gerarda Cappuccio, Annalaura Torella, et al.
JIMD Reports
|
March 11, 2020
The c.1A > C start codon mutation in <i>CLN3</i> is associated with a protracted disease course
Willemijn F E Kuper, Claudia van Alfen, Linda van Eck, et al.
Page
of 125
Search research articles
Search
Showing results (781-790 of 1,250) with videos related to
Sort By:
Page
of 125
JIMD Reports
|
November 18, 2020
Fabry disease in India: A multicenter study of the clinical and mutation spectrum in 54 patients
Sheela Nampoothiri, Dhanya Yesodharan, Amrita Bhattacherjee, et al.
JIMD Reports
|
November 18, 2020
Cerebrotendinous xanthomatosis-associated diarrhea and response to chenodeoxycholic acid treatment
Eric P Brass, Bianca M L Stelten, Aad Verrips
JIMD Reports
|
February 20, 2020
Early detection of lysosomal diseases by screening of cases of idiopathic splenomegaly and/or thrombocytopenia with a next-generation sequencing gene panel
Gloria Muñoz, David García-Seisdedos, Crina Ciubotariu, et al.
JIMD Reports
|
February 20, 2020
NGLY1 deficiency: Novel patient, review of the literature and diagnostic algorithm
Patryk Lipiński, Anna Bogdańska, Agnieszka Różdżyńska-Świątkowska, et al.
JIMD Reports
|
February 20, 2020
Neurotoxicity including posterior reversible encephalopathy syndrome after initiation of calcineurin inhibitors in transplanted methylmalonic acidemia patients: Two case reports and review of the literature
Femke Molema, Monique Williams, Janneke Langendonk, et al.
JIMD Reports
|
March 11, 2018
Cognitive and Behavioural Outcomes of Paediatric Liver Transplantation for Ornithine Transcarbamylase Deficiency
Louise Crowe, Vicki Anderson, Winita Hardikar, et al.
JIMD Reports
|
January 31, 2018
Beneficial Effect of BH<sub>4</sub> Treatment in a 15-Year-Old Boy with Biallelic Mutations in DNAJC12
Monique G M de Sain-van der Velden, Willemijn F E Kuper, Marie-Anne Kuijper, et al.
JIMD Reports
|
December 16, 2017
Expert Opinion vs Patient Perspective in Treatment of Rare Disorders: Tooth Removal in Lesch-Nyhan Disease as an Example
Adam C Cotton, R B Bell, H A Jinnah
JIMD Reports
|
March 11, 2020
Cavitating and tigroid-like leukoencephalopathy in a case of <i>NDUFA2</i>-related disorder
Marianna Alagia, Gerarda Cappuccio, Annalaura Torella, et al.
JIMD Reports
|
March 11, 2020
The c.1A > C start codon mutation in <i>CLN3</i> is associated with a protracted disease course
Willemijn F E Kuper, Claudia van Alfen, Linda van Eck, et al.
Page
of 125