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Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene, Treatment of epidermolysis bullosa
Case Medical Research
◽
10.31525/cmr-1e0edb1
◽
2019
◽
Author(s):
Keyword(s):
Epidermolysis Bullosa
◽
Antisense Oligonucleotide
◽
Orphan Designation
◽
Gene Treatment
◽
Col7a1 Gene
Download Full-text
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Orphan designation: Adeno-associated viral vector serotype 2 containing the human REP1 gene, Treatment of choroideraemia
Case Medical Research
◽
10.31525/cmr-399699
◽
2019
◽
Author(s):
Keyword(s):
Viral Vector
◽
Orphan Designation
◽
Gene Treatment
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene, Treatment of spinal muscular atrophy
Case Medical Research
◽
10.31525/cmr-1e4edc3
◽
2019
◽
Author(s):
Keyword(s):
Spinal Muscular Atrophy
◽
Viral Vector
◽
Muscular Atrophy
◽
Orphan Designation
◽
Gene Treatment
◽
Smn Gene
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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
◽
Serotype 5
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Orphan designation: Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene, Treatment of leber's congenital amaurosis
Case Medical Research
◽
10.31525/cmr-12e1a9a
◽
2019
◽
Author(s):
Keyword(s):
Viral Vector
◽
Orphan Designation
◽
Gene Treatment
◽
Leber’S Congenital Amaurosis
Download Full-text
Orphan designation: Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene, Treatment of Fabry disease
Case Medical Research
◽
10.31525/cmr-128cc2f
◽
2019
◽
Author(s):
Keyword(s):
Fabry Disease
◽
Viral Vector
◽
Orphan Designation
◽
Gene Treatment
◽
Alpha Galactosidase
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Orphan designation: Diacerein, Treatment of epidermolysis bullosa
Case Medical Research
◽
10.31525/cmr-152a1a1
◽
2019
◽
Author(s):
Keyword(s):
Epidermolysis Bullosa
◽
Orphan Designation
Download Full-text
Orphan designation: Adeno-associated viral vector serotype 8 containing the human MTM1 gene, Treatment of X-linked myotubular myopathy
Case Medical Research
◽
10.31525/cmr-10e0aa5
◽
2019
◽
Author(s):
Keyword(s):
Viral Vector
◽
Myotubular Myopathy
◽
Orphan Designation
◽
Gene Treatment
Download Full-text
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
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10.31525/cmr-3996ed
◽
2018
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Author(s):
Keyword(s):
Factor Viii
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Viral Vector
◽
Coagulation Factor
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Coagulation Factor Viii
◽
Haemophilia A
◽
Factor Viii Gene
◽
Orphan Designation
◽
Gene Treatment
◽
Serotype 5
Download Full-text
Orphan designation: adeno-associated viral vector serotype hu68 containing the human SMN1 gene, Treatment of spinal muscular atrophy
Case Medical Research
◽
10.31525/cmr-6939f8
◽
2018
◽
Author(s):
Keyword(s):
Spinal Muscular Atrophy
◽
Viral Vector
◽
Muscular Atrophy
◽
Smn1 Gene
◽
Orphan Designation
◽
Gene Treatment
Download Full-text
Orphan designation: losartan, Treatment of epidermolysis bullosa
Case Medical Research
◽
10.31525/cmr-fb98a4
◽
2019
◽
Author(s):
Keyword(s):
Epidermolysis Bullosa
◽
Orphan Designation
◽
Losartan Treatment
Download Full-text
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