Industrializing rare disease therapy discovery and development

Nature Biotechnology

Volumn 35, Issue 2, 2017, Pages 117-118

  Ekins, Sean ^a,b,c  

^a Collaborations Pharmaceuticals Inc ^*  (United States)

^b Phoenix Nest   (United States)

^c Hereditary Neuropathy Foundation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS 9; ANIMAL MODEL; CELL AGGREGATION; CELL STRUCTURE; CLINICAL TRIAL (TOPIC); ENZYME REPLACEMENT; GENE THERAPY; HEALTH CARE COST; HUMAN; IMMUNOGENICITY; IMMUNOLOGY; INDUSTRIALIZATION; INTERMEDIATE FILAMENT; LETTER; NONHUMAN; PERIPHERAL NEUROPATHY; PRIORITY JOURNAL; PROTEIN FOLDING; RARE DISEASE; SANFILIPPO SYNDROME; DRUG DEVELOPMENT; DRUG INDUSTRY; GIANT AXONAL NEUROPATHY;

CLINICAL TRIALS AS TOPIC; DRUG DISCOVERY; DRUG INDUSTRY; GENETIC THERAPY; GIANT AXONAL NEUROPATHY; HUMANS; RARE DISEASES;

EID: 85012119105     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.3787     Document Type: Letter

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