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PubPharm (15)
1
Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids
enthalten in:
European journal of neurology
| 2024
von
Schiava, M.
|
Lofra, R.
|
Bourke, J.
| +20
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2
Gene therapy in Duchenne muscular dystrophy : Identifying and preparing for the challenges ahead
enthalten in:
Neuromuscular disorders : NMD
| 2021
von
Heslop, E.
|
Turner, C.
|
Irvin, A.
| +4
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3
Abrupt warming and salinification of intermediate waters interplays with decline of deep convection in the Northwestern Mediterranean Sea
enthalten in:
Scientific reports
| 2020
von
Margirier, F.
|
Testor, P.
|
Heslop, E.
| +12
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4
The RD-Connect Registry & Biobank Finder : a tool for sharing aggregated data and metadata among rare disease researchers
enthalten in:
European journal of human genetics : EJHG
| 2018
von
Gainotti, S.
|
Torreri, P.
|
Wang, C.
| +18
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5
The TREAT-NMD advisory committee for therapeutics (TACT) : an innovative de-risking model to foster orphan drug development
enthalten in:
Orphanet journal of rare diseases
| 2015
von
Heslop, E.
|
Csimma, C.
|
Straub, V.
| +85
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6
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development
enthalten in:
Orphanet journal of rare diseases
| 2015
von
Heslop, E.
|
Csimma, C.
|
Straub, V.
| +12
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7
RD-Connect : an integrated platform connecting databases, registries, biobanks and clinical bioinformatics for rare disease research
enthalten in:
Journal of general internal medicine
| 2014
von
Thompson, R.
|
Johnston, L.
|
Taruscio, D.
| +16
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8
RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research
enthalten in:
Journal of general internal medicine
| 2014
von
Thompson, R.
|
Johnston, L.
|
Taruscio, D.
| +16
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9
RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research
enthalten in:
Journal of general internal medicine
| 2014
von
Thompson, R.
|
Johnston, L.
|
Taruscio, D.
| +16
Wird geladen...
10
RD-Connect: An Integrated Platform Connecting Databases, Registries, Biobanks and Clinical Bioinformatics for Rare Disease Research
enthalten in:
Journal of general internal medicine
| 2014
von
Thompson, R.
|
Johnston, L.
|
Taruscio, D.
| +16
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1
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4
Journal of general internal medicine
2
Neuromuscular disorders
2
Neuromuscular disorders : NMD
2
Orphanet journal of rare diseases
1
European journal of human genetics : EJHG
1
European journal of neurology
1
Human mutation
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Scientific reports
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8
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6
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3
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2
Duchenne muscular dystrophy
1
Barriers
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1
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DMD
1
De-risking
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Drug development
1
EK scale in adults with DMD
1
Gene therapy
1
Neuromuscular disease
1
Oligonucleotides, Antisense
1
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1
Research Support, U.S. Gov't, Non-P.H.S.
1
TREAT-NMD
1
cardiac function in adults with DMD
1
disease registries
1
glucocorticoid dose in adults with DMD
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2020-
12
2010-2019
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