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1. Association Between Lysine Reduction Therapies and Cognitive Outcomes in Patients With Pyridoxine-Dependent Epilepsy

2. Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study

4. Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial

5. Infants with Tyrosinemia Type 1: Should phenylalanine be supplemented?

7. Eye movement disorders in inborn errors of metabolism – a quantitative analysis of 37 patients

8. Timing of therapy and neurodevelopmental outcomes in 18 families with pyridoxine-dependent epilepsy

13. Homozygous UBA5 Variant Leads to Hypomyelination with Thalamic Involvement and Axonal Neuropathy

15. Consensus guidelines for the diagnosis and management of pyridoxine‐dependent epilepsy due to α‐aminoadipic semialdehyde dehydrogenase deficiency

18. A Recurrent De Novo PACS2 Heterozygous Missense Variant Causes Neonatal-Onset Developmental Epileptic Encephalopathy, Facial Dysmorphism, and Cerebellar Dysgenesis (vol 102, pg 995, 2018)

20. A Recurrent De Novo PACS2 Heterozygous Missense Variant Causes Neonatal-Onset Developmental Epileptic Encephalopathy, Facial Dysmorphism, and Cerebellar Dysgenesis

21. Molybdenum cofactor deficiency type A: Prenatal monitoring using MRI

22. A Recurrent De Novo PACS2 Heterozygous Missense Variant Causes Neonatal-Onset Developmental Epileptic Encephalopathy, Facial Dysmorphism, and Cerebellar Dysgenesis

23. Paediatric motor phenotypes in early-onset ataxia, developmental coordination disorder, and central hypotonia.

27. Heterogeneous clinical spectrum of DNAJC12-deficient hyperphenylalaninemia: from attention deficit to severe dystonia and intellectual disability

30. The Burke-Fahn-Marsden Dystonia Rating Scale is Age-Dependent in Healthy Children

31. Infants with Tyrosinemia Type 1:Should phenylalanine be supplemented?

32. Heterogeneous clinical spectrum of DNAJC12-deficient hyperphenylalaninemia: from attention deficit to severe dystonia and intellectual disability.

34. Reliability and discriminant validity of ataxia rating scales in early onset ataxia.

37. Favorable Outcome in a Newborn With Molybdenum Cofactor Type A Deficiency Treated With cPMP

39. Favorable Outcome in a Newborn With Molybdenum Cofactor Type A Deficiency Treated With cPMP

43. Clinical and biochemical spectrum of D-bifunctional protein deficiency

45. NUBPLmutations in patients with complex I deficiency and a distinct MRI pattern

48. Consensus guidelines for the diagnosis and management of pyridoxine-dependent epilepsy due to α-aminoadipic semialdehyde dehydrogenase deficiency.

49. A Recurrent De Novo PACS2 Heterozygous Missense Variant Causes Neonatal-Onset Developmental Epileptic Encephalopathy, Facial Dysmorphism, and Cerebellar Dysgenesis.

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