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Last Updated : 06/20/2022

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Found 7 publications

Towards Precision Therapies for Inherited Disorders of Neurodegeneration with Brain Iron Accumulation.

Journal: Tremor and other hyperkinetic movements (New York, N.Y.)
Published: December 15, 2021
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Summary: Towards Precision Therapies for Inherited Disorders of Neurodegeneration with Brain Iron Accumulation.

NBIA Syndromes: A Step Forward from the Previous Knowledge.

Journal: Neurology India
Published: November 08, 2021
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Summary: NBIA Syndromes: A Step Forward from the Previous Knowledge.

Down regulation of the expression of mitochondrial phosphopantetheinyl-proteins in pantothenate kinase-associated neurodegeneration: pathophysiological consequences and therapeutic perspectives.

Journal: Orphanet journal of rare diseases
Published: May 06, 2021
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Summary: Down regulation of the expression of mitochondrial phosphopantetheinyl-proteins in pantothenate kinase-associated neurodegeneration: pathophysiological consequences and therapeutic perspectives.

Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study.

Condition: Pantothenate Kinase-associated Neurodegeneration
Journal: Orphanet journal of rare diseases
Treatment Used: Pantethine
Number of Patients: 15
Published: September 15, 2020
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Summary: Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study.

Management of rare movement disorders in Europe: outcome of surveys of the European Reference Network for Rare Neurological Diseases.

Condition: Rare Movement Disorders in Europe
Journal: European journal of neurology
Treatment Used: Botulinum Toxin or Deep Brain Stimulation
Number of Patients: 0
Published: May 10, 2020
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Summary: Management of rare movement disorders in Europe: outcome of surveys of the European Reference Network for Rare Neurological Diseases.

Palliative care in 9 children with neurodegeneration with brain iron accumulation.

Condition: Children with neurodegeneration with brain iron accumulation
Journal: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
Treatment Used: Palliative care
Number of Patients: 9
Published: November 24, 2019
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Summary: Palliative care in 9 children with neurodegeneration with brain iron accumulation.

Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.

Condition: Pantothenate Kinase-Associated Neurodegeneration
Journal: The Lancet. Neurology
Treatment Used: Deferiprone
Number of Patients: 100
Published: June 17, 2019
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Summary: Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.
Showing 1-7 of 7

Last Updated : 06/20/2022