Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Nature Reviews Drug Discovery

Volumn 19, Issue 2, 2020, Pages 93-111

  Tambuyzer, Erik ^a,b   Vandendriessche, Benjamin ^c,d   Austin, Christopher P ^e   Brooks, Philip J ^e   Larsson, Kristina ^f   Miller Needleman, Katherine I ^g   Valentine, James ^h   Davies, Kay ⁱ   Groft, Stephen C ^e   Preti, Robert ^j   Oprea, Tudor I ^k,l   Prunotto, Marco ^m  

^a BioPontis Alliance for Rare Diseases Foundation fup son   (Belgium)

^b BioPontis Alliance Rare Disease Foundation   (United States)

^c Byteflies   (Belgium)

^d Case Western Reserve University   (United States)

^e NATIONAL INSTITUTES OF HEALTH   (United States)

^f EUROPEAN MEDICINES AGENCY   (United Kingdom)

^g CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^h Hyman Phelps & McNamara PC   (United States)

ⁱ UNIVERSITY OF OXFORD   (United Kingdom)

^j Showa Denko Materials Co   (United States)

^k UNIVERSITY OF NEW MEXICO SCHOOL OF MEDICINE   (United States)

^l UNIVERSITY OF NEW MEXICO   (United States)

^m UNIVERSITY OF GENEVA   (Switzerland)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA 1 ANTITRYPSIN; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY CD3; OLIGONUCLEOTIDE; ORPHAN DRUG; RETROVIRUS VECTOR; SMALL INTERFERING RNA; SMALL MOLECULE TRANSPORT AGENT;

ANTIBODY ENGINEERING; CELL THERAPY; DISEASE EXACERBATION; DRUG APPROVAL; DRUG LEGISLATION; DRUG MANUFACTURE; DRUG MECHANISM; DRUG REPOSITIONING; DRUG TARGETING; EMPHYSEMA; ENZYME REPLACEMENT; GENE THERAPY; GRAFT REJECTION; HEMOPHILIA A; HEMOPHILIA B; HUMAN; IMMUNOTHERAPY; MEDICAL RESEARCH; MEDICAL TECHNOLOGY; PATIENT DECISION MAKING; PATIENT PARTICIPATION; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; RARE DISEASE; REVIEW; SUBSTITUTION THERAPY; VIRAL GENE DELIVERY SYSTEM; DRUG DEVELOPMENT;

DRUG APPROVAL; DRUG DEVELOPMENT; DRUG REPOSITIONING; HUMANS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 85076831788     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/s41573-019-0049-9     Document Type: Review

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