Orphan designation: Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein, Treatment of Friedreich's ataxia

Case Medical Research ◽

10.31525/cmr-6ac6fb ◽

2018 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Orphan Designation ◽

Protein Treatment

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Orphan designation: Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein, Treatment of Friedreich's ataxia

Case Medical Research ◽

10.31525/cmr-67955b ◽

2018 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Orphan Designation ◽

Protein Treatment

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Orphan designation: Adenovirus-associated viral vector serotype 5 containing the human RPGR gene, Treatment of retinitis pigmentosa

Case Medical Research ◽

10.31525/cmr-12e1a9d ◽

2019 ◽

Author(s):

Keyword(s):

Retinitis Pigmentosa ◽

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene, Treatment of haemophilia A

Case Medical Research ◽

10.31525/cmr-3996ed ◽

2018 ◽

Author(s):

Keyword(s):

Factor Viii ◽

Viral Vector ◽

Coagulation Factor ◽

Coagulation Factor Viii ◽

Haemophilia A ◽

Factor Viii Gene ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: leriglitazone, Treatment of Friedreich’s ataxia

Case Medical Research ◽

10.31525/cmr-277ad3b ◽

2020 ◽

Author(s):

Keyword(s):

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Orphan Designation

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Orphan designation: Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene, Treatment of leber's congenital amaurosis

Case Medical Research ◽

10.31525/cmr-178003a ◽

2019 ◽

Author(s):

Keyword(s):

Staphylococcus Aureus ◽

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Leber’S Congenital Amaurosis

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Orphan designation: Adenovirus associated viral vector serotype 5 containing the human RPE65 gene, Treatment of leber's congenital amaurosis

Case Medical Research ◽

10.31525/cmr-12e1a94 ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Leber’S Congenital Amaurosis

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Orphan designation: 4-hydroxy-6-{2-[4-(trifluoromethyl)phenyl]ethyl}pyridazin-3(2H)-one, Treatment of Friedreich’s ataxia

Case Medical Research ◽

10.31525/cmr-12043f1 ◽

2019 ◽

Author(s):

Keyword(s):

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Orphan Designation

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Cardiomyopathy in Friedreich's ataxia: assessment by echocardiography and cardiac magnetic resonance imaging

Aktuelle Neurologie ◽

10.1055/s-2005-919549 ◽

2005 ◽

Vol 32 (S 4) ◽

Author(s):

C Meyer ◽

G Schmid ◽

S Görlitz ◽

M Schillings ◽

I Wilhelm ◽

...

Keyword(s):

Magnetic Resonance Imaging ◽

Cardiac Magnetic Resonance ◽

Magnetic Resonance ◽

Cardiac Magnetic Resonance Imaging ◽

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Resonance Imaging

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Friedreich’s ataxia (FA), comorbidities and anaesthetic management.

10.26226/morressier.58f5b02fd462b80296c9e1f8 ◽

2017 ◽

Author(s):

Gilda F. Pardey Bracho

Keyword(s):

Anaesthetic Management ◽

Friedreich’S Ataxia ◽

Friedreich's Ataxia

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Otoneurological Findings in Friedreich's Ataxia and Other Inherited Neuropathies

International Journal of Audiology ◽

10.3109/00206098609078373 ◽

1986 ◽

Vol 25 (2) ◽

pp. 84-91 ◽

Author(s):

E. Cassandro ◽

F. Mosca ◽

L. Sequino ◽

F. A. De Falco ◽

G. Campanella

Keyword(s):

Friedreich’S Ataxia ◽

Friedreich's Ataxia ◽

Inherited Neuropathies

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