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PubPharm (41)
1
SUCCESSES AND PITFALLS IN ORPHAN DRUG DEVELOPMENT FOR SICKLE CELL DISEASE
enthalten in:
Blood advances
| 2024
von
Costa, E.
|
Isgrò, A.
|
de Montalembert, M.
| +3
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2
Advancing rare disease treatment : EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
enthalten in:
Gene therapy
| 2024
von
Kalland, M.
|
Pose-Boirazian, T.
|
Palomo, G.
| +10
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3
Uptake of orphan drugs in the WHO essential medicines lists
enthalten in:
Bulletin of the World Health Organization
| 2024
von
Costa, E.
|
Moja, L.
|
Wirtz, V.
| +4
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4
Adoptive cell therapy for treatment of rare diseases and orphan designation.
enthalten in:
ResearchSquare.com
| 2023
von
Kalland, M.
|
Pose, T.
|
Palomo, G.
| +10
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5
The European landscape for gene therapies in orphan diseases : 6-year experience with the EMA Committee for Orphan Medicinal Products
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2023
von
Palomo, G.
|
Pose-Boirazian, T.
|
Naumann-Winter, F.
| +10
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6
Traits, trends and hits of orphan drug designations in cystic fibrosis
enthalten in:
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
| 2023
von
Costa, E.
|
Girotti, S.
|
van den Ham, H.
| +4
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7
X-ray polarization evidence for a 200-year-old flare of Sgr A
enthalten in:
Nature
| 2023
von
Marin, F.
|
Churazov, E.
|
Khabibullin, I.
| +98
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8
Vela pulsar wind nebula X-rays are polarized to near the synchrotron limit
enthalten in:
Nature
| 2022
von
Xie, F.
|
Di Marco, A.
|
La Monaca, F.
| +90
CommentIn: Nature. 2022 Dec;612(7941):641-642. - PMID 36543948
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9
Trends and determinants of epinephrine prescriptions : a proxy of anaphylaxis epidemiology?
enthalten in:
Current opinion in allergy and clinical immunology
| 2022
von
Costa, E.
|
Tanno, L.
|
Salazzari, D.
| +3
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10
Polarized blazar X-rays imply particle acceleration in shocks
enthalten in:
Nature
| 2022
von
Liodakis, I.
|
Marscher, A.
|
Agudo, I.
| +98
CommentIn: Nature. 2022 Nov;611(7937):673-674. - PMID 36418446
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