Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale

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Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale

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wetenschappelijk artikel

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наукова стаття, опублікована у 2017

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Development of the Impact of J ...... y on Physical Activities scale

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Development of the Impact of J ...... y on Physical Activities scale

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Development of the Impact of J ...... y on Physical Activities scale

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Development of the Impact of J ...... y on Physical Activities scale

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Development of the Impact of J ...... y on Physical Activities scale

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Development of the Impact of J ...... y on Physical Activities scale

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P1476

Development of the Impact of J ...... y on Physical Activities scale

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Linda S Deal

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Sheri E Fehnel

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Susan Martin

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T Michelle Brown

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P2860

Developing therapeutic approaches for metachromatic leukodystrophy

Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance

Language and cognition in children with metachromatic leukodystrophy: onset and natural course in a nationwide cohort.

The frequency of lysosomal storage diseases in The Netherlands.

Population carrier rates of pathogenic ARSA gene mutations: is metachromatic leukodystrophy underdiagnosed?

Metachromatic leukodystrophy: a case of triplets with the late infantile variant and a systematic review of the literature.

Lysosomal storage diseases: diagnostic confirmation and management of presymptomatic individuals.

Patient-reported outcomes to support medical product labeling claims: FDA perspective.

Developing treatment options for metachromatic leukodystrophy.

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options.

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scholarly article

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10.1186/S41687-018-0041-X

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English

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P478

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2017-01-01T00:00:00Z

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29757307

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5934920

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