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PubPharm (46)
1
The value of knowing : preferences for genetic testing to diagnose rare muscle diseases
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Mansfield, C.
|
Boeri, M.
|
Coulter, J.
| +4
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2
The value of knowing: preferences for genetic testing to diagnose rare muscle diseases
enthalten in:
Orphanet journal of rare diseases
| 2024
von
Mansfield, C.
|
Boeri, M.
|
Coulter, J.
| +4
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3
Pharmacokinetics of Alglucosidase Alfa Manufactured at the 4000-L Scale in Participants with Pompe Disease : A Phase 3/4 Open-Label Study
enthalten in:
Clinical pharmacology in drug development
| 2023
von
Nicolas, X.
|
Hurbin, F.
|
Periquet, M.
| +4
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4
Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study
enthalten in:
Neurology. Clinical practice
| 2023
von
Dimachkie, M.
|
Kishnani, P.
|
Ivanescu, C.
| +10
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5
COMPOSITIONS AND METHODS FOR TREATING LATE-ONSET POMPE DISEASE
enthalten in:
Europäisches Patentamt
| 2023
von
FINN PATRICK
|
HAMED ALAA
|
AN HAACK KRISTINA
| +1
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6
COMPOSITIONS AND METHODS FOR TREATING LATE-ONSET POMPE DISEASE
enthalten in:
Europäisches Patentamt
| 2023
von
FINN PATRICK
|
HAMED ALAA
|
AN HAACK KRISTINA
| +1
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7
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks : A Phase 3 Randomized Clinical Trial
enthalten in:
JAMA neurology
| 2023
von
Kishnani, P.
|
Diaz-Manera, J.
|
Toscano, A.
| +125
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8
COMPOSITIONS AND METHODS FOR TREATING LATE-ONSET POMPE DISEASE
enthalten in:
Europäisches Patentamt
| 2023
von
FINN PATRICK
|
HAMED ALAA
|
AN HAACK KRISTINA
| +1
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9
후기 발병형 폼페병을 치료하기 위한 조성물 및 방법
enthalten in:
Europäisches Patentamt
| 2023
von
FINN PATRICK
|
HAMED ALAA
|
AN HAACK KRISTINA
| +1
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10
Compositions and methods for treating late-onset pompe disease
enthalten in:
Europäisches Patentamt
| 2023
von
FINN PATRICK
|
HAMED ALAA
|
AN HAACK KRISTINA
| +1
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che
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2020-
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2010-2019
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