Orphan designation: Recombinant human acid ceramidase, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-1f6761d ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Acid Ceramidase ◽

Orphan Designation ◽

Human Acid

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Orphan designation: Recombinant human acid ceramidase, Treatment of Farber disease

Case Medical Research ◽

10.31525/cmr-1f67617 ◽

2019 ◽

Author(s):

Keyword(s):

Acid Ceramidase ◽

Farber Disease ◽

Orphan Designation ◽

Human Acid

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Orphan designation: Sinapultide, dipalmitoylphosphatidylcholine palmitoyl-oleoyl phosphatidylglycerol, sodium salt and palmitic acid, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-134bca6 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Palmitic Acid ◽

Sodium Salt ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: Alpha-1 antitrypsin (inhalation use), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-110cf5b ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Alpha 1 Antitrypsin

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Orphan designation: Alpha-1 antitrypsin (inhalation use), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-1103412 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Alpha 1 Antitrypsin

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Orphan designation: Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-22295fd ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Cholesterol Treatment

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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor), Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-58722a ◽

2018 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation ◽

Tert Butyl

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Orphan designation: Mannitolum, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-99e07f ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation

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Orphan designation: 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-bc10f3 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Benzoic Acid ◽

Acid Treatment ◽

Orphan Designation

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Orphan designation: 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate, Treatment of cystic fibrosis

Case Medical Research ◽

10.31525/cmr-8789ef ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Orphan Designation

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Orphan designation: Amikacin sulfate (liposomal), Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis

Case Medical Research ◽

10.31525/cmr-de5510 ◽

2019 ◽

Author(s):

Keyword(s):

Cystic Fibrosis ◽

Pseudomonas Aeruginosa ◽

Lung Infection ◽

Orphan Designation ◽

Amikacin Sulfate

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