Research ExplorerScientific StaffMiriam Nickel

ID: 64097

Miriam Nickel

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Affiliations
ORCID: 0000-0002-1870-1142

Publications

Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Language Delay in Patients with CLN2 Disease: Could It Support Earlier Diagnosis?

Research output: SCORING: Contribution to journal › Short publication › Research › peer-review

Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease)

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Visual perception and macular integrity in non-classical CLN2 disease

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Natural history of MRI brain volumes in patients with neuronal ceroid lipofuscinosis 3: a sensitive imaging biomarker

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

The Unified Batten Disease Rating Scale (UBDRS): Validation and reliability in an independent CLN3 disease sample

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Natural History Studies in NCL and Their Expanding Role in Drug Development: Experiences From CLN2 Disease and Relevance for Clinical Trials

Research output: SCORING: Contribution to journal › SCORING: Review article › Research

Development of the "Hamburg Best Practice Guidelines for ICV-Enzyme Replacement therapy (ERT) in CLN2 Disease" Based on 6 Years Treatment Experience in 48 Patients

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Management of CLN1 Disease: International Clinical Consensus

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Investigating health-related quality of life in rare diseases:a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

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